CELEBRATION, Fla., Feb. 04, 2026 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with ...
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FDA accepts Beren Therapeutics’ NDA for Niemann-Pick disease
The submission includes data indicating a meaningful survival benefit for infantile-onset NPC patients treated with adrabetadex.
To start patients on Miplyffa, prescribers can fill out an enrollment form on Miplyffa.com. Miplyffa™ (arimoclomol) is now available for use in combination with miglustat for the treatment of ...
Beren Therapeutics P.B.C.®, the parent company of Mandos LLC® and leader in cholesterol trafficking biology and cyclodextrin-based therapeutics, today announced that the U.S. Food and Drug ...
Zevra Therapeutics, Inc. announced the publication of long-term data on the efficacy and safety of MIPLYFFA® (arimoclomol) for treating Niemann-Pick disease type C (NPC) in the journal Molecular ...
New data from pre-specified analysis shows patients on concomitant miglustat who switched from placebo to MIPLYFFA experienced a decline in annual disease progression Nomination for Best Poster Award ...
First patient dosed in global pivotal clinical trial program for late infantile/juvenile onset NPC and GM1/GM2 gangliosidoses. Both studies are part of Azafaros’ mission to develop treatments for the ...
Several rare disease patient populations received their first-ever FDA-approved drug since Rare Disease Day last year, signifying progress in closing treatment gaps for rare disease. Since Rare ...
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