- 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 .

This is the first of (hopefully!) many blog posts to come. Who am I? My name is Ellie. I have two cousins who were diagnosed with Niemann-Pick Type C Disease (NP-C) as teenagers. I have worked as a carer for people with NP-C and am on the Science Advisory Board for the Australian Niemann-Pick Type C Disease Foundation (ANPDF). I am a student finishing off my Masters degree in Genomics and Health. My studies include a range of topics including rare genetic conditions, types of genetic testing, and screening measures. My passion is learning more about people and what makes us who we are. I hope that in my future career, I can make science topics interesting and easy to understand for everyone! Why a blog? The motivation behind starting this blog was to help talk about ideas, considerations, and approaches to coping with NP-C as an individual, family, and community. I will be including scientific research and concepts in my posts, so everything that I say (unless otherwise stated) is backed up with evidence. I would love for people to engage with these posts – so any feedback is welcome. My question to you I need your help in discovering what topics need a voice. What do you want me to talk about or see on this blog? ANPDF is about the whole of the NP-C community – researchers, families, clinicians, and more. I want to do my best to write blogs that appeal to the needs and/or interests of the readers. Want to know some special tips for NDIS funding? Ideas for wheelchair-friendly activities in Melbourne? How to take care of yourself whilst you take care of others? Let me know! Stay supportive and supported, Ellie

Read the letter from Mandos Health here: Here is a summary of the article: INPDA 2023 Meeting : Mandos attended the 2023 INPDA meeting in Tarragona, Spain, highlighting progress in the NPC community and supporting adrabetadex (VTS-270) research . Upcoming Conferences : Mandos will sponsor the 2024 Parseghian Fund Annual Conference and the NNPDF Family Support & Medical Conference . Research and Development : the FDA has accepted Mandos' request for a Type C Meeting scheduled in October to discuss the direction of the adrabetadex program’s development. Expanded Access Program: the EAP age criteria have been expanded to include patients from 3 months to 25 years old 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 .

Summaries of latest research advances related to Niemann-Pick diseases, acid sphingomyelinase deficiency (ASMD) and Niemann-Pick type C disease (NPCD), based on selected peer-reviewed publications in scientific journals. By Frank W. Pfrieger, PhD Institute of Cellular and Integrative Neurosciences Centre National de la Recherche Scientifique / University of Strasbourg Strasbourg, France Read the full article here: These summaries provide a glimpse into the diverse research efforts aimed at understanding and addressing NPCD and ASMD, shedding light on biomarkers, genetic modifiers, diagnosis, treatment approaches, and potential therapeutic targets. Please feel free to distribute and forward this issue, and to send feedback to: fw-pfrieger@gmx.de or frank.pfrieger@unistra.fr Support research for a cure of Niemann-Pick disease type C in Australia: Donate online   Shop at our online NPC store   Fundraise your way! Check our free resources and inspo online

We are thrilled to announce the results of our recent fundraiser for Kando Martial Arts! Thanks to the incredible support from our community, we have raised a total of $1,603 to support ANPDF projects like the National Care and Guidelines. Thank you to everyone who participated and contributed to the success of our fundraiser. Your generosity will help us continue to provide quality martial arts training and 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, Kando Martials Arts Rowville and to all our event sponsors and contributor: McKays Family Butchers STUD PARK, Danika William-Diaz, Frisco the Label, Coles Rowville, Woolworths Rowville, Bakers Delight Rowville.

**New raffle prizes added! Are you ready to enhance your self-defence skills and boost your confidence? Look no further because Shihan Paul's Self-Defence class is here to empower you! Teaming up with ANPDF-volunteer, Ellie Van Velsen, this dynamic duo is ready to guide you through an exhilarating journey of self-discovery and protection. Self-defence is not just about physical techniques; it's about equipping yourself with the mindset and skills needed to navigate challenging situations confidently. Shihan Paul, a seasoned expert in martial arts, brings a wealth of knowledge and experience to every class. His enthusiasm is contagious, making each session not only educational but also incredibly fun! Meet Your Instructors: Paul: With years of experience in various martial arts disciplines, Paul is dedicated to sharing his expertise with individuals looking to build their self-defence skills. His passion for teaching is evident in his enthusiastic approach, creating a supportive environment for students to thrive. Ellie: As an ANPDF-volunteer, Ellie brings a unique perspective to the self-defence classes. Her commitment to empowering individuals and promoting safety is truly inspiring. Ellie's presence adds a valuable dimension to the classes, ensuring that participants receive a well-rounded and comprehensive training experience. What to Expect: 1.5hr Self-Defence Class: Learn effective self-defence techniques that are easy to remember and can be applied in real-life scenarios. ANPDF Kiosk: Selling Sausages in Bread ($3.50), Cold and Hot Drinks ($2.50), and more. Limited Raffle: Pre-purchase your raffle tickets at the event checkout! Each raffle ticket is just $5 to go in the draw to win one of six awesome prizes with a total prize pool over $1500! Raffle prizes - Tickets are $5 each First Prize: Frisco the Label Family Pack valued at $700rrp. Second Prize: Clock Artwork valued at $490rrp. Third prize: Handmade Resin Cheeseboard Set valued at $130rrp. Fourth prize: $100 KMA Gift Voucher. Fifth prize: $100 KMA Gift Voucher. Sixth prize: Lindt chocolate box valued at $26rrp. Event Schedule: Day: Saturday, 24th August 2024 Time: Stalls open at 11am until 4:30pm, Class starts at 2:00pm Location: Shop 5, Stud Park Shopping Centre, Rowville Frequently Asked Questions I'm not very sporty or coordinated, will that be a problem? Not at all. In fact, many students starting in our adult martial arts classes would describe themselves like that before they started. So, you will fit right in! You’ll find that our very experienced martial arts Instructor Shihan Paul, is trained to keep his teaching simple. So even the most uncoordinated person will develop their self-defence skills! Come see for yourself! What should I wear and bring? Make sure you wear comfortable clothes you can do sport in! Not really suitable to practice kicks in a skirt.😳 Be sure to bring a water bottle to the self-defence class, or you can buy water from our kiosk. Where should I park? Kando Martial Arts is located in shop 5 of the Stud Park Shopping Centre, so there should be plenty of parking in the parking lot. Is the location and class wheelchair accessible? Yes! Kando Martial Arts Rowville is a wheelchair accessible venue and has a wheelchair accessible parking lot. Due to the requirements of the matting during the Self-Defence Class, we are limited to five (5) wheelchair-abled attendees. Please select the wheelchair accessible ticket at checkout to ensure we are able to accommodate all attendees. Join Us Today! Whether you're a complete beginner or have some experience in self-defence, Shihan Paul's class offers something for everyone. Come and discover the warrior within you under the guidance of seasoned instructors who are passionate about your safety and well-being. Don't miss out on this incredible opportunity to transform yourself mentally and physically. Join us at Shihan Paul's Self-Defence class and embark on a journey of empowerment and self-discovery! Remember, your safety is your superpower. Let's unlock it together with Shihan Paul and Ellie Van Velsen! By embracing self-defence techniques, you're not just learning to protect yourself physically; you're also cultivating mental resilience and confidence. Take the first step towards empowerment today!

FDA Advisory Committee Votes Favourably that the Data Support Arimoclomol as Effective Treatment for Patients with Niemann-Pick Disease Type C Read the letter from Zevra Therapeutics here: Here is a summary of the article: FDA Advisory Committee Vote: The U.S. FDA Genetic Metabolic Diseases Advisory Committee (GeMDAC) voted favourably (11 yes, 5 no) that Arimoclomol is effective in treating patients with NPC. Clinical Data Support: The committee considered clinical data from pivotal trials, long-term open-label extension studies, and expanded access programs. About NPC: NPC is an ultra-rare, progressive, neurodegenerative lysosomal storage disorder caused by mutations in NPC1 or NPC2 genes. Arimoclomol: Zevra’s investigational drug candidate for NPC has received Orphan Drug designation and other regulatory designations (2). The FDA has set a Prescription Drug User Fee Act (PDUFA) action date of September 21, 2024 (3,4). 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.