Regulatory Review of Santhera's Idebenone by the CHMP
Santhera announced today that the EMA’s CHMP has granted a requested extension of the clock-stop in the regulatory procedure for…
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NS Pharma Announces Publication of Clinical Trial Data for Viltolarsen
NS Pharma, a wholly owned subsidiary of Nippon Shinyaku, announced that JAMA Neurology has published results from a clinical trial…
Santhera completes enrollment of Phase 3 SIDEROS Study with Puldysa® (Idebenone)
Today, Santhera Pharmaceuticals announced full recruitment of its Phase 3 SIDEROS study with idebenone in Duchenne muscular dystrophy (DMD). The…
Updated clinical data on Pfizer's phase 1b gene therapy study
Pfizer today announced updated clinical data on PF-06939926, an investigational gene therapy being developed to treat Duchenne muscular dystrophy (DMD).…
Capricor announces HOPE-2 Study results
The 12-month data from HOPE-2 showed statistically meaningful improvements in the PUL 2.0 in CAP-1002 treated patients (p=0.05) with a…
Catabasis: update on edasalonexent
Catabasis shared today their Catabasis Connection newsletter, with updates on edasalonexent and information shared at the MDA Virtual Poster Session.…
UPDATE ON CLINICAL HOLD IGNITE DMD
This morning, Solid Biosciences issued a press release following receipt of written communication from the FDA regarding the clinical hold placed…
Italfarmaco update on Givinostat
Update on advanced-stage clinical development program with Givinostat. The Italfarmaco Group provided today an update on the clinical development program…
Marketing authorization in Japan of VILTEPSO®
NS Pharma announced today that the Ministry of Health, Labour and Welfare (MHLW) has approved VILTEPSO® Intravenous Infusion 250 mg…
NDA accepted for filing by the FDA for antisense oligonucleotide Viltolarsen
NS Pharma announced the FDA has filed a New Drug Application (NDA) for their antisense oligonucleotide drug NS-065/NCNP-01 (viltolarsen). Viltolarsen is…
PTC Therapeutics publicises real-world data Translarna (ataluren)
PTC Therapeutics Announces First Publication of Real-World Data Showing TranslarnaTM (ataluren) Significantly Preserves Ability to Walk for Longer in Children…
Roche provided rights to Sarepta’s investigational gene therapy for Duchenne.
Roche and Sarepta Therapeutics signed a licensing agreement providing Roche commercial rights to SRP-9001, Sarepta’s investigational gene therapy for Duchenne. …
FDA approves VYONDYS 53TM (golodirsen) in the US
Sarepta Therapeutics Announces FDA Approval of VYONDYS 53TM (golodirsen) Injection for the Treatment of Duchenne Muscular Dystrophy (DMD) in Patients…
VISION-DMD Working Group Meeting
Athens, 6 and 8 December 2019 – The Vision-DMD Working Group Meeting organized by the World Duchenne Organization, preceded the…
Solid Biosciences IGNITE DMD placed on clinical hold
Official press release CAMBRIDGE, Mass., Nov. 12, 2019 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) today provided a clinical…
Roche terminates SPITFIRE trial
Unfortunately we have a very sad piece of news today: after an interim analysis of the SPITFIRE trial with Roche’s…
Webinar on PolarisDMD: Phase 3 Clinical Trial of Edasalonexent
CAMBRIDGE, Mass., November 2, 2018 - Catabasis Pharmaceuticals, Inc (NASDAQ:CATB), a clinical-stage biopharmaceutical company, and Parent Project Muscular Dystrophy (PPMD) will host a webinar:…
Sarepta announces clinical hold lifted for its Duchenne Muscular Dystrophy micro-dystrophin gene therapy program
Sarepta Therapeutics, Inc., announced that the Food and Drug Administration (FDA) has lifted the clinical hold for the Company’s Duchenne…
Catabasis announces initiation of Phase 3 PolarisDMD trial for edasalonexent
Catabasis Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, today announced the initiation of PolarisDMD, the Company’s Phase 3 trial for edasalonexent…
Sarepta receives negative CHMP re-examination opinion for eteplirsen
This morning, Sarepta announced that following its request for re-examination, the Committee for Human Medicinal Products (CHMP) in Europe issued…
Pfizer terminates clinical studies of Domagrozumab
Pfizer Inc. announced on August 30, 2018 that it is terminating two ongoing clinical studies evaluating domagrozumab (PF-06252616) for the treatment of…
