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Meizon has been granted both Orphan Drug Designation & Rare Pediatric Disease designation by the US FDA. Meizon is developing a portfolio of patented biologically active molecules for preventing, treating and potentially reversing the trajectory of brain disorders. DL33 is a therapeutic treatment for Dementia by replenishing deficient DL33 levels in the brain. On November 5, Meizon Innovations announced that the U.S. FDA has granted its drug DL33 both Orphan Drug Designation and Rare Pediatric Disease Designation for Niemann-Pick disease type C. These designations recognise DL33's potential to address significant unmet needs. Unlike other treatments, Meizon's approach is rooted in scientific evidence aimed at proving DL33's effectiveness in treating dementia. Initial studies in mice have shown promise in reversing dementia effects, but further research is needed to meet FDA standards. Next steps include animal toxicology testing, followed by human safety testing in a Phase 1 study. What is DL33? To join our fight, as well as fund vital research for a cure, support the Australian NPC Disease Foundation in its goals of "Research. Cure. Persevere." Ways to support NP-C in Australia: Donate online Shop at our online NPC store Fundraise your way! Check our free resources and inspo online .

Raised $2,130.00 Details Date/Time: October 1st to 30th, 2024 Draw Date and Time: Thursday 31st of October 2024 at 12:00 PM Location: Drawn online via rafflys Organiser: Deanna Carpino States available: VIC, SA, QLD, NSW, and TAS Terms and Conditions Terms and Conditions apply. This raffle is being held for Australian NPC Disease Foundation Inc ABN: 87767010514, c/- 3 Waller Street, Benalla, VIC, 3672. The Raffle commences on the 1st of October 2024, closes on the 30th of October 2024 and will be drawn on the 31st of October 2024 online at 12 pm. The draw will be conducted using App-Sorteos.com, an online tool. Ticket buyers do not have to be present at the draw. Winners will be contacted by email within 2 days after draw. Winners may also receive a phone call from the raffle organisers. Details of the Winner(s) will be displayed live at the Minds in Motion Charity Cocktail and displayed online at www.npcd.org.au. State(s) where tickets are on sale VIC. Persons under the age of 18 years are unable to enter. #SilverRaffle #raffle FIRST PRIZE: PS5 VIrtual Racing Simulator Bundle Pack ($5,000 rrp) PlayStation 5 Slim Console F1 24 - PS5 Game 6 Playseat Trophy Racing Chair Playseat Trophy Gearshift & Handbrake Holder Logitech G29 Driving Force Racing Wheel Logitech G Driving Force Shifter Winners: 1ST PRIZE WINNER S. Previti, Ticket Number 2WVH-ZTV1-VBC1R-54 (VIC) Thank you to our sponsors! Donation of first prize Goat Club Interested in organising your own raffle or fundraiser? This raffle was conducted both online and in-person.   Contact  us today for free advice and resources including how-to guides.

On October 18th, students at Alexandra Primary School unleashed their creativity for a special "Crazy Hair Day" fundraiser, all in support of Niemann-Pick Awareness Month! The event encouraged students to bring a gold coin donation in exchange for showcasing their wildest, wackiest hairstyles, transforming the school grounds into a sea of colorful, spiky, and unique hairdos. Organised by Carly, a dedicated ANPDF committee member and mum, this fundraiser not only allowed students to have some lighthearted fun but also served a meaningful purpose. Through the students' generous contributions, Alexandra Primary School raised $136 , which will help support the mission of ANPDF to spread awareness and offer support for those impacted by Niemann-Pick Disease. Niemann-Pick Disease is a rare, genetic condition, and initiatives like Crazy Hair Day are vital to shining a light on this lesser-known illness. The silver awareness ribbon for Niemann-Pick Disease symbolises the resilience of families and communities united in this cause, and Alexandra Primary School's Crazy Hair Day was a beautiful display of support. Thank you to all the students, teachers, and families who participated, and especially to Carly for organizing this fun and impactful event! Together, we’re making a difference, one crazy hairstyle at a time! Let's keep up the great work and continue to support our Niemann-Pick community! Special Thanks to the Alexandra Primary School for supporting the Niemann-Pick Community in Australia. Check out this beautiful school online here: Alexandra Primary School or join their conversation on facebook

We are thrilled to announce the results of our recent fundraiser: Poker Tournament for Niemann Pick! Thanks to the incredible support from the Benalla and wider Victorian community, we have raised a total of $2,300 to support research, families and projects for Niemann-Pick. 🏆 1st Prize Winner and last Man standing: Rob Mundy 🏆 2nd Prize: Les Cairns 3rd Prize: Damien Broughton 4th Prize: Tony Chetcuti 5th Prize: Jordan Smith Thank you to everyone who participated and contributed to the success of our fundraiser. Your generosity will help us continue to provide quality programs for our members. Let's keep up the great work and continue to support our Niemann-Pick community! Special Thanks to our Venue Partner, Benalla Bowls Club and to all our event sponsors and contributors: Benalla Golf Shop, Benalla Printing, Fruit n fare, Benalla Myotherapy, Mind body safe yoga, Benalla taxi group, Terry White Chemist, Paul Robinson, and Rob Mundy.

Read the letter from Mandos Health here: The Mandos Health October 2024 update highlights their progress in Niemann-Pick Type C (NPC) therapies. Key points include a productive FDA meeting on October 10, involving key stakeholders like Dr. Berry-Kravis and Dr. Porter. Mandos also celebrated NPC Awareness Month through community engagement and attended major summits like the NORD Breakthrough Summit. The Expanded Access Program continues to support NPC patients and their families. They remain committed to developing science-driven solutions for NPC. To join our fight, as well as fund vital research for a cure, support the Australian NPC Disease Foundation in its goals of "Research. Cure. Persevere." Ways to support NP-C in Australia: Donate online Shop at our online NPC store Fundraise your way! Check our free resources and inspo online .

As an organisation passionately dedicated to spreading awareness about Niemann-Pick Disease, we are thrilled to delve into the significance behind the emblematic silver ribbon and its role in advocating for those impacted by this rare and challenging condition. Brace yourselves for an enlightening journey into the history, symbolism, and profound importance of raising awareness for Niemann-Pick Disease! Jump to: Symbolism 🎗️ History 🎗️ Importance 🎗️ Advocacy Unveiling the Silver Ribbon: A Symbol of Hope and Unity The silver ribbon stands as a beacon of solidarity, embodying support, empathy, and encouragement for individuals battling Niemann-Pick Disease. Its shimmering hue symbolizes resilience, hope, and clarity amid the shadows of this debilitating illness. By proudly donning the silver ribbon, we not only show our support for those fighting this rare disorder but also broadcast a powerful message of unity and strength in our collective mission to raise awareness. The Noble Legacy of Awareness Ribbons: A Golden Thread of Advocacy The tradition of awareness ribbons traces back to a heartwarming act of compassion in the early 20th century. What began as a simple yellow ribbon tied in remembrance evolved into a universal language of support for various causes and conditions. The silver ribbon, specifically dedicated to Niemann-Pick Disease, continues this legacy by uniting individuals worldwide in a shared commitment to advocacy and understanding. Shining Light on Niemann-Pick Disease: Why Awareness Matters Niemann-Pick Disease, a rare genetic disorder impacting lipid metabolism, presents unique challenges for those affected and their families. By raising awareness through initiatives like the #SilverLight campaign, we illuminate this lesser-known condition, offering hope, resources, and a sense of community to those navigating the complexities of the disease. Education and awareness are pivotal in fostering early detection, research funding, and, most importantly, support for individuals living with Niemann-Pick Disease. A Call to Action: Empowering Change Through Advocacy As health professionals, families, and compassionate individuals, we wield the power to effect change through advocacy and awareness. By championing the silver ribbon as a symbol of unity and understanding, we amplify the voices of those touched by Niemann-Pick Disease, sparking conversations, driving research, and nurturing a community of care and support. Together, we can make a difference, one silver ribbon at a time. Join the Movement: Shine a Silver Light on the Fight Against Niemann-Pick Disease In conclusion, the silver ribbon serves as a powerful emblem of hope, empathy, and advocacy for individuals grappling with Niemann-Pick Disease. Its radiant symbolism transcends borders, languages, and differences, uniting us in a common cause to raise awareness, foster support, and ignite change. Let us embrace the silver ribbon as a beacon of light in the darkness, a symbol of strength in the face of adversity, and a testament to the unbreakable spirit of those touched by Niemann-Pick Disease. Together, we can wear our silver ribbons as badges of honour, as symbols of unity, and as beacons of hope for a future where Niemann-Pick Disease is not just a challenge but a conquered chapter in the book of human resilience. Let us rise, shine, and illuminate the path forward for a brighter, more informed tomorrow. Stay silver-strong, dear advocates! As you embark on your journey to raise awareness for Niemann-Pick Disease, remember that every silver ribbon you wear carries the weight of hope, the power of unity, and the promise of a brighter future for all those impacted by this challenging condition. Wear it with pride, share its story with passion, and let its message resonate far and wide. Together, we can make a difference—one ribbon, one voice, one heart at a time!

- By Deanna Carpino (ANPDF), Tuesday October 15, 2024 Life has a way of surprising us when we least expect it. For one family, a pivotal moment came when their beloved son Greg was diagnosed with Niemann-Pick Type C (NPC) Disease at the age of 39. This rare and devastating genetic disorder drastically altered the course of their lives, but through the storm, they found something stronger than the challenges they faced—each other. The family had three children, two of whom were affected by NPC: Greg and his sister, who was also diagnosed at 39 in 2020. A Diagnosis That Changed Everything Greg’s journey began innocently, with small moments of forgetfulness. His family attributed his memory lapses to the hits he took during his rugby league days. But as these instances grew more frequent, concern deepened, and so began the long, arduous road to diagnosis. After seven months of tests (including a skin biopsy), setbacks, and endless waiting, their fears were confirmed—Greg had Niemann-Pick disease type C (NPC), a diagnosis that would forever change their lives. “We noticed that Greg had difficulty remembering things and suspected that he had one too many knocks to the head when playing rugby league," recalls his mother, Maz. As Greg’s mother reflects, her words carry the weight of love and sacrifice. She recalls those early days—how her heart ached with worry, how the paperwork and medical appointments seemed endless, and yet she pressed on, even as she faced her own battles. "During that time, I had gone through two bouts of cancer, a hip replacement, and chemotherapy. It left me with neuropathy in my hands and feet.” Despite the diagnosis, Greg’s courage shone through. He continued working at The Star Casino, holding on to his independence for as long as he could. But by 2018, the disease’s cruel progression made it impossible for him to continue. His father, who had already retired, stepped in to care for Greg full-time, and by 2019, Maz too retired, dedicating every ounce of her energy to her son’s well-being. A Daily Fight for Greg’s Quality of Life Now in his late 40s, Greg’s battle with NPC has brought on symptoms of dementia, short-term memory loss, and ataxia, among other difficulties. Though unable to participate in drug trials, his family focused on making sure his life remained meaningful. “We engaged a Support Coordinator, Occupational Therapist, Speech Pathologist and Support Workers who ensured that he was showered and got out into the community instead of being stuck in front of a TV all day,” his mother explains. Healthcare providers have played a pivotal role in Greg’s care. From the initial diagnosis to ongoing support, the family’s interactions with doctors, neurologists, metabolic geneticists, and various therapists have been instrumental. The collaboration between healthcare professionals and the family has ensured that Greg receives comprehensive care tailored to his needs. For Greg’s aging parents, caring for him has been an all-encompassing responsibility, one that challenges their strength each day. But despite their own limitations, they’ve navigated the complexities of the system, from NDIS to support coordinators, always with Greg’s best interests in mind. In July 2023, Greg moved into the Sunnyfield group home, and in May 2024, he transitioned to the Windgap group home closer to his parents, making it easier for the family to visit frequently. Each visit is a testament to their unwavering love. A Future Reimagined “When I retired, I envisaged going on holidays around Australia, joining a local sewing group and enjoying my passion for sewing and quilting, but this was not possible,” Greg’s mother reflects. Retirement for Greg’s parents didn’t bring the adventures they had once envisioned. There were no trips around Australia or quiet afternoons spent pursuing hobbies. Instead, their lives revolved around Greg. Yet in their sacrifices, they found unexpected joy and purpose. Greg’s mother now uses her sewing skills to raise funds for the ANPDF and create dementia fidget mats for those in need. A Community That Lifts Them Up Through the toughest of times, Greg’s family found comfort in something they hadn’t anticipated—community. Connecting with other NPC families and meeting Professor Mark Walterfang in Melbourne opened doors to both knowledge and emotional support. The NPC Foundation became their lifeline, offering them solidarity and understanding. Now, Maz serves on the committee for the foundation, finding strength in helping others who walk the same path. “What I do have is an incredible group of friends who are there to support me when needed,” she says. “None of this is easy, but I have the most incredible friends and an NPC community that’s always there for me,” she says with gratitude. The monthly Zoom meetings have become a source of solace, a reminder that they are never alone in this fight. Hope and Resilience: One Day at a Time Though they cannot change Greg’s diagnosis, they can ensure his days are filled with care and love. Their hope lies in future treatments, not only for Greg but also for others like his sister, and for families just beginning their journeys with NPC. Maz knows that while the road is hard, they are making a difference—not just in Greg’s life, but in the lives of others facing similar battles. “We can only be there for him and ensure he has everything he needs,” she says softly. “I am hopeful for the younger ones and the treatments available, but for us it is planning for the future and just taking it one day at a time.” In the end, Greg’s story is not just one of struggle but of immense love, resilience, and the power of a family determined to stand together through it all. They continue to make a difference in Greg’s and his sister’s lives, as well as in the lives of others affected by NPC.

15 October 2024 - INPDR, Tyne & Wear England. We are thrilled to share the announcement of a significant development in the fight against Niemann-Pick Disease! A new research site dedicated to Niemann-Pick is set to open in Melbourne, Australia. This initiative is a collaborative effort aimed at advancing our understanding of this rare disease and improving treatment options for those affected. Key Highlights of the New Site Collaboration with Leading Experts : This site will bring together some of the most respected researchers in the field, fostering an environment of innovation and progress. Focus on Patient-Centric Research : The research conducted at this new facility will prioritise the needs and experiences of patients, ensuring that their voices are heard in the development of new therapies. Expanding Global Research Network : This new site is part of a broader initiative to establish a global network of research facilities dedicated to Niemann-Pick Disease, enhancing collaboration and resource sharing. What This Means for the Community The opening of this new research site is a beacon of hope for individuals and families affected by Niemann-Pick Disease. It represents a commitment to finding effective treatments and ultimately a cure for this challenging condition. We believe that this advancement will contribute to significant breakthroughs in understanding Niemann-Pick Disease and improving the lives of those living with it. Mandy Whitechurch , founder and president of the  Australian NPC Disease Foundation Inc  (ANPDF), shared: “I am so proud and thrilled to see the Royal Melbourne Hospital as our first Australian INPDR registered site. Heartfelt thanks to Profressor Mark Walterfang and Courtney for all the efforts put in to get this established..."  Join Us in Celebrating This Milestone We encourage our community to share this exciting news and continue supporting research efforts. Together, we can make a difference in the lives of those impacted by Niemann-Pick Disease. For more details, read the full press release here . Together, we can push forward in our mission to combat Niemann-Pick Disease!

NP-C Corner Special Edition by Dave Badcock, Melbourne University Student I was shocked. Sitting at the Australian NPC Conference this year, I overheard a parent ask why her family was not encouraged to speak to a genetic counsellor during her child’s NPC diagnosis journey. I thought: “How could they not be supported? Do people (professionals and the community) really know what a genetic counsellor is, or what they do?” I would like to share what I know so that the NPC community is aware of these incredible people doing incredible work. My name is Dave, and I am a current Genetic Counselling student volunteering with the ANPDF. Throughout the process of testing, countless appointments, confusing words, new doctors, drug names and more, it’s safe to say the journey of diagnosis can be a really challenging time. I like to think of the role of a genetic counsellor as making this journey a little less complicated. Whether helping to clarify your understanding on a condition, to assisting with health services or treatment. Genetics counsellors can assist you with many of the factors that make diagnoses so tough. They are there to listen to your thoughts and feelings, because they know how challenging a diagnosis and treatment journey can be. So, what really is a genetic counsellor you may ask? Well, they often work in hospitals, but they’re not doctors, they’re also not psychologists. They are qualified counsellors, who have also been trained in genetics. Equipped with both a genetics background and counselling skills, they facilitate informed decision-making. They are there to listen and support you in the choices you make. Lisette Curnow (a practicing genetic counsellor) gave an insightful presentation at the 2024 Australian NP-C Conference in Melbourne. Lisette’s talk was focused on stripping back some of the terminology and science into understandable language. She looked at how NP-C is inherited, how it affects families and some strategies around communicating within a family. Speaking to members of the audience, they appreciated how straightforward Lisette spoke. They respected how the information was relevant for them and that she made the time to answer people’s questions… the perfect example of a genetic counsellor!    But what does the community think about genetic counsellors?  I recently spoke with Deanna Carpino, the General Manager of the ANPDF and a proud NP-C mum of two. She talked positively about her experience with genetic counsellors when undertaking her boys’ diagnoses. Deanna spoke about their patience and use of relatable language.  “Leaving me feeling empowered to make informed decisions” about the genetics of Niemann-Pick Disease, future medical appointments and what to expect on journey to having a second child. I admit I may be biased; however, I think genetic counsellors occupy an invaluable role in the ever-evolving healthcare system. I want the NP-C community to know genetic counsellors are here to help. Want to get in touch? Ask your doctor to get you in contact and we will do our best. Dave References: https://pixabay.com/illustrations/counselor-welfare-strengths-6741671/ https://pixabay.com/illustrations/therapy-psychologist-waiting-room-7540958/

October is a significant month for raising awareness about these rare and devastating conditions. We come together to shine a silver light on two critical disorders: Niemann-Pick disease type C (NP-C) and Acid Sphingomyelinase Deficiency (ASMD). In this article: Understanding NP-C | Understanding ASMD | NPD + the Silver Ribbon | Get Involved | Resources Understanding NP-C NP-C is a neurologically degenerative disorder characterised by the accumulation of lipids (fats) within cells. It affects both children and adults. The average time to diagnose NP-C is five years , emphasizing the need for early detection and increased awareness. Families dealing with NP-C face immense challenges as their loved ones gradually lose essential abilities Learn more Understanding ASMD ASMD, previously known as Niemann-Pick disease type A and B , is caused by a deficiency of the enzyme acid sphingomyelinase . It leads to the accumulation of sphingomyelin in various organs, affecting the liver, spleen, lungs, and brain. ASMD has a wide spectrum of severity, from early infantile forms (type A) to later-onset forms (type B). Learn more Niemann-Pick Disease and the Silver Ribbon The silver awareness ribbon represents solidarity with individuals and families impacted by NP. By wearing or displaying this ribbon, we raise awareness about NP and advocate for early detection, research, and support. During Niemann-Pick Awareness Month , which takes place in October , we come together to educate others, promote understanding, and emphasize the urgent need for effective treatments and a cure. How You Can Get Involved Spread Awareness : Share information about NPD on social media using the hashtags #NPDawareness and #SilverLight . Encourage conversations and help others recognise the signs and symptoms. Support Research : Consider donating to organisations like the Australian NPC Disease Foundation Inc (ANPDF) , which funds critical research at The Florey Institute of Neuroscience and Mental Health . Research holds the key to finding effective treatments and ultimately a cure. Participate : Attend NPD awareness events , engage in community activities, and stay informed about advancements in NP research. Learn more Together, let’s shine a silver light on NPD and make a difference! Join us to raise awareness🎗for NPC! Edit and download customised social media profile images Edit and/or download read-to-go social media awareness posts Like, follow and share from our social media channels Facebook @NiemannPickOz Instagram @NiemannPickOz Twitter @NiemannPickOz LinkedIn /anpdf Print a downloadable poster below for your local shop or workplace Donate online to help us fund ground-breaking research or financially supporting families left in the NDIS gap Buy a raffle ticket or Nominate the ANPDF in your own fundraiser

IntraBio Inc is very excited to confirm the U.S. Food and Drug Administration (FDA) has approved AQNEURSA (levacetylleucine) for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg. Quick Facts: AQNEURSA was approved by the FDA on September 24, 2024, marking it as the second treatment for NPC approved within a week, following the approval of Miplyffa. AQNEURSA is indicated for the treatment of neurological symptoms associated with Niemann-Pick disease type C (NPC) in both adults and pediatric patients who weigh at least 15 kgs. AQNEURSA targets and corrects the genetic mutations responsible for Niemann-Pick disease type C (NPC), helping to reduce lipid accumulation in cells and slow disease progression. On September 24, 2024, the FDA approved AQNEURSA, marking the second treatment for Niemann-Pick disease type C (NPC) approved within a week. This rapid succession of approvals, including Miplyffa earlier in the week, underscores the FDA’s commitment to addressing rare diseases and providing new therapeutic options for patients. This breakthrough is particularly impactful for Australian families, who have long awaited advancements in treatment options. In their announcement, the FDA stated, “This is the second treatment the FDA has approved for NPC within the span of a week. Today’s action further underscores the agency’s commitment to support development of new treatments for rare diseases,” said Janet Maynard, M.D., M.H.S., director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine, in the FDA’s Center for Drug Evaluation and Research. “This approval again demonstrates the FDA’s commitment to work with the scientific community to overcome the unique challenges that may arise with rare disease drug development.” What is AQNEURSA? AQNEURSA (levacetylleucine, IB1001) is a novel therapy designed to address the underlying causes of NPC. It works by targeting and correcting the genetic mutations responsible for the disease, potentially slowing its progression and improving the quality of life for patients. How Does AQNEURSA Work? AQNEURSA is designed to address the underlying causes of NPC by targeting the genetic mutations responsible for the disease. These mutations affect the body's ability to metabolize lipids, leading to their accumulation in cells. AQNEURSA works by correcting these faulty cellular processes, thereby reducing lipid buildup. This action helps slow the progression of the disease, potentially allowing patients to maintain their abilities and independence for a longer period. By improving cellular function, AQNEURSA aims to enhance the overall quality of life for patients and their families, offering hope and a better future for those affected by NPC. What is the prescribing information for AQNEURSA? AQNEURSA (levacetylleucine) is an oral medication approved for the treatment of neurological symptoms associated with Niemann-Pick disease type C (NPC) in adults and paediatric patients weighing at least 15 kilograms. Here is a summary of the prescribing information: Dosage and Administration Administration: Orally up to three times per day, with or without food. Dosage:  The recommended dose varies depending on the individual's body weight. Specific dosing guidelines are provided in the prescribing information. Safety Information Pregnancy Warning:  AQNEURSA may cause embryo-fetal harm if used during pregnancy. Females should inform their healthcare provider of a known or suspected pregnancy before taking AQNEURSA. Common Side Effects:  The most common side effects include abdominal pain, difficulty swallowing, upper respiratory tract infections, and vomiting. Clinical Study Study Design:  The safety and efficacy of AQNEURSA were evaluated in a randomized, double-blind, placebo-controlled, two-period, 24-week crossover study involving 60 patients aged 4 years and older with confirmed NPC and at least mild disease-related neurological symptoms. Primary Efficacy Outcome:  The primary efficacy outcome was measured using a modified version of the Scale for the Assessment and Rating of Ataxia (SARA), referred to as the functional SARA (fSARA). Participants treated with AQNEURSA for 12 weeks showed better outcomes in the fSARA score compared to when they were treated with placebo¹². For more detailed information, including specific dosing guidelines and comprehensive safety information, please refer to the official prescribing information provided by the FDA ¹². What Does This Mean for NP-C Families in Australia? The FDA approval of AQNEURSA is a significant milestone, but there are still a few steps before it becomes widely available in Australia. As with Miplyffa, the ANPDF will actively advocate for the swift approval and accessibility of AQNEURSA in our country. Advocacy will be crucial as we take the first steps in joining efforts to urge the Therapeutic Goods Administration (TGA) to expedite the approval process for these treatments. Together, we can push for progress and support those affected by Niemann-Pick disease. Stay connected with ANPDF for updates and to participate in advocacy efforts to bring these treatments to Australia. Looking Ahead The FDA approval of AQNEURSA is a monumental step forward in the fight against Niemann-Pick disease. For Australian families, it brings hope, potential access to advanced treatment, and a renewed sense of purpose in advocating for a brighter future. Together, we can continue to push for progress and support those affected by this challenging condition. Together, we can make a difference. To join our fight, as well as fund vital research for a cure, support the Australian NPC Disease Foundation in its goals of "Research. Cure. Persevere." Ways to support NP-C in Australia: Donate online Check out upcoming events Shop at our online NPC store Fundraise your way! Check our free resources and inspo online . Sources: (1) FDA Approves New Drug to Treat Niemann-Pick Disease, Type C. https://www.fda.gov/news-events/press-announcements/fda-approves-new-drug-treat-niemann-pick-disease-type-c (2) FDA Approves New Drug to Treat Niemann-Pick Disease, Type C - PR Newswire. https://www.prnewswire.com/news-releases/fda-approves-new-drug-to-treat-niemann-pick-disease-type-c-302257740.html

Quick Facts: MIPLYFFA is the first FDA-approved treatment for Niemann-Pick disease type C (NPC), an ultra-rare and progressive neurodegenerative disease MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of NPC in adults and pediatric patients 2 years of age and older Zevra receives rare pediatric disease priority review voucher in conjunction with approval. As parents of children diagnosed with Niemann-Pick disease type C (NP-C), we understand the daily challenges and emotional rollercoaster that come with this journey. Today, we bring you a beacon of hope: the recent approval of Miplyffa (arimoclomol) by the U.S. Food and Drug Administration (FDA) as the first-ever treatment for NP-C . What is Miplyffa? Miplyffa is an oral medication designed to treat the neurological symptoms associated with NP-C. It works in combination with miglustat, another drug that helps manage the disease. This combination has shown promising results in slowing down the progression of NP-C, offering a new avenue of hope for families affected by this rare genetic disorder ¹ How Does Miplyffa Work? Miplyffa targets the cellular stress response, helping cells to function more effectively despite the genetic mutations that cause NP-C. In clinical trials, patients treated with Miplyffa experienced a slower progression of symptoms, particularly in areas such as mobility, speech, swallowing, and fine motor skills ¹ . This means that children and adults with NP-C can potentially enjoy a better quality of life for a longer period. What Does This Mean for NP-C Families in Australia? While Miplyffa has been approved by the FDA, it is essential for Australian families to stay informed about its availability and approval status within Australia. The Australian NPC Disease Foundation (ANPDF) is actively working to advocate for the swift approval and accessibility of Miplyffa in our country. We encourage you to stay connected with ANPDF for updates and to participate in advocacy efforts to bring this treatment to Australia. The journey with NP-C is undoubtedly challenging, but as a community, we can support each other through these times. Looking Ahead The approval of Miplyffa marks a significant milestone in the fight against NP-C. It represents a step forward in providing our children with the treatments they deserve. Let us continue to support each other, stay informed, and advocate for the best possible care for our loved ones. Together, we can make a difference. To join our fight, as well as fund vital research for a cure, support the Australian NPC Disease Foundation in its goals of "Research. Cure. Persevere." Ways to support NP-C in Australia: Donate online Check out upcoming events Shop at our online NPC store Fundraise your way! Check our free resources and inspo online .