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Cyclo Therapeutics Community Update | June 18, 2025
June 18, 2025 – Newark, NJ Rafael Holdings has announced that an independent Data Monitoring Committee (DMC) has completed its scheduled...
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Jun 202 min read
Pfrieger's Digest | Issue 12 - August 2024 – December 2024
Summaries of latest research advances related to Niemann-Pick diseases, acid sphingomyelinase deficiency (ASMD) and Niemann-Pick type C...
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Jan 211 min read
Azafaros Community Update | January 9, 2025
Exciting Advances in Azafaros Clinical Programs for NPC and GM Gangliosidoses Azafaros has announced significant progress toward global...
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Jan 101 min read
Azafaros Community Update | December 20, 2024
Exciting Advances in Azafaros Clinical Programs for NPC and GM Gangliosidoses Today, Azafaros shared an important update about their...
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Dec 20, 20241 min read
Meizon Innovations Community Update | November 5, 2024
Meizon has been granted both Orphan Drug Designation & Rare Pediatric Disease designation by the US FDA.
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Nov 6, 20241 min read
Mandos Health Media Release | October 22, 2024
Read the letter from Mandos Health here: The Mandos Health October 2024 update highlights their progress in Niemann-Pick Type C (NPC)...
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Oct 23, 20241 min read
Exciting News: New INPDR Site Opening in Melbourne for Niemann-Pick Disease
15 October 2024 - INPDR, Tyne & Wear England. We are thrilled to share the announcement of a significant development in the fight against...
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Oct 14, 20242 min read
Second treatment approved by FDA for Niemann-Pick disease, type C: Introducing AQNEURSA
FDA approval of AQNEURSA offers new hope for Australian families with Niemann-Pick disease, promising better treatment and quality of life.
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Sep 25, 20244 min read
A New Hope for NP-C Families: Introducing Miplyffa
New Hope for NP-C Families: Miplyffa, the first FDA-approved treatment for NPC, offers a promising future for our loved ones. Stay informed!
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Sep 21, 20242 min read
Mandos Health Media Release | September 10, 2024
Read the letter from Mandos Health here: Here is a summary of the article: INPDA 2023 Meeting : Mandos attended the 2023 INPDA meeting in...
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Sep 16, 20241 min read
Pfrieger's Digest | Issue 11 - March 2024 – July 2024
Summaries of latest research advances related to Niemann-Pick diseases, acid sphingomyelinase deficiency (ASMD) and Niemann-Pick type C...
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Sep 6, 20241 min read
Zevra Therapeutics Media Release | August 2, 2024
FDA Advisory Committee Votes Favourably that the Data Support Arimoclomol as Effective Treatment for Patients with NP-C
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Aug 7, 20241 min read
Azafaros Media Release | July 16, 2024
Azafaros announces positive topline Phase 2 study data with nizubaglustat in GM2 gangliosidosis and Niemann-Pick disease type C Read the...
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Jul 17, 20241 min read
Cyclo Therapeutics Media Release | May 30, 2024
Cyclo Therapeutics Achieves Landmark Milestone with Completion of Enrollment of Last Patient in Phase 3 Pivotal TransportNPC™ Trial of...
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Jun 1, 20241 min read
IntraBio Media Release | March 26, 2024
IntraBio Announces U.S. FDA Accepts New Drug Application for IB1001 for the Treatment of Niemann-Pick disease type C Read the full...
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Mar 27, 20241 min read
Azafaros Media Release | March 12, 2024
Azafaros announces completion of 12-week Phase 2 RAINBOW study evaluating lead asset nizubaglustat in rare disease patients.
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Mar 14, 20241 min read
Zevra Therapeutics Media Release | March 4, 2024
Zevra Therapeutics Provides FDA Update On The PDUFA Action Date For Arimoclomol As A Treatment For Niemann-Pick Disease Type C Read the...
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Mar 5, 20241 min read
IntraBio Media Release | February 7, 2024
The New England Journal of Medicine has published a "Science behind the study" editorial on IB1001 Read the full announcement from...
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Feb 8, 20241 min read
IntraBio Media Release | February 7, 2024
The New England Journal of Medicine has published the detailed results of the IB1001-301 Phase 3, Pivotal study with N-acetyl-L-leucine...
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Feb 3, 20241 min read
IntraBio Community Update | February 1, 2024
New Drug Application for IB1001 for the treatment of NP-C disease was submitted to the US FDA in January 2024 Read the full statement...
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Feb 1, 20241 min read