Business Wire

Positive Niemann-Pick disease type C (NPC) and GM2 gangliosidosis data from nizubaglustat Phase 2 RAINBOW study conducted by Azafaros presented at major metabolic disease ...

LEIDEN, Netherlands--(BUSINESS WIRE)--Azafaros has announced that data from the ongoing double-blind, placebo-controlled Phase 2 RAINBOW study investigating its lead asset, nizubaglustat in patients ...
Business Wire

International Niemann-Pick Disease Registry and Mandos Health announce new research collaboration using real-world data to evaluate the treatment effects of VTS-270 to manage ...

TYNE AND WEAR, England--(BUSINESS WIRE)--The International Niemann-Pick Disease Registry (INPDR) and biopharmaceutical company Mandos Health today announce a new collaboration giving Mandos Health ...

Zevra Therapeutics Announces Publication of Positive MIPLYFFA® Data from Pediatric Substudy in Infants with Niemann-Pick Disease Type C (NPC)

Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on bringing life-changing therapeutics to people living with rare diseases, announced the ...
Medical Xpress

FDA approves Miplyffa for treatment of Niemann-Pick disease, type C

The U.S. Food and Drug Administration has approved Zevra Therapeutics' Miplyffa (arimoclomol), an oral medication for the treatment of Niemann-Pick disease, type C (NPC). Miplyffa, in combination with ...
Sunderland Echo on MSN

Wearside charity and its fightback from a devastating fire is spotlighted in new documentary film

‘We provide a sense of community to those affected by these devastating rare conditions’ ...
Nasdaq

Zevra Therapeutics Celebrates and Supports Global Niemann-Pick Disease Awareness Day on October 19th and Niemann-Pick Disease Awareness Month Throughout October

CELEBRATION, Fla., Oct. 19, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA), a rare disease therapeutics company, proudly supports the Niemann-Pick community on Global Niemann-Pick ...

Apertura Gene Therapy Announces Cooperative Research and Development Agreement with Institutes of the NIH to Test Investigational Gene Therapy for Niem…

Apertura Gene Therapy, a biotechnology company developing next-generation AAV capsids for delivering genetic medicines, today ...
Medical Xpress

Treatment with N-acetyl-L-leucine beneficial for Niemann-Pick disease

Treatment with N-acetyl-L-leucine (NALL) for 12 weeks yields better neurological status than placebo among patients with genetically confirmed Niemann-Pick disease type C, according to a study ...
Nasdaq

Zevra Therapeutics Showcases MIPLYFFA® Data at National Niemann Pick Disease Foundation Conference

Zevra Therapeutics presents MIPLYFFA data at the NNPDF Conference, showcasing its FDA-approved treatment for Niemann-Pick Disease type C. Zevra Therapeutics, Inc. announced that its therapy MIPLYFFA® ...
Morningstar

THX Pharma and Biocodex Announce a Strategic Licensing Agreement in Batten, Gaucher and Niemann-Pick Type C Diseases | Morningstar

THX Pharma (Theranexus) and Biocodex, an independent international pharmaceutical group, today announced the execution of a strategic licensing agreement to advance the development of two drug ...
News Medical

Many individuals with Niemann-Pick disease lack neutralizing antibodies against AAV2 or AAV9

A new study in the peer-reviewed journal Human Gene Therapy showed that more than half of individuals with Niemann-Pick disease, type C1 (NPC1) who were tested lacked neutralizing antibodies against ...
Healio

FDA approves second Niemann-Pick disease type C treatment

Please provide your email address to receive an email when new articles are posted on . The FDA has approved Aqneursa as an oral treatment for neurological symptoms associated with Niemann-Pick ...