Specialty Pharma Journal Specialty, Precision & Technology
CAMBRIDGE, Mass., Dec. 20, 2018 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a leader in precision genetic medicine for rare diseases, announced today that it has completed the submission of its rolling New Drug Application (NDA) seeking accelerated approval for golodirsen (SRP-4053), a phosphordiamidate morpholino oligimer engineered to treat those …
Tag Archives: duchenne muscular dystrophy
December, 2018
November, 2018
-
20 November
Santhera Enters into Agreement to Acquire Option from Idorsia for Exclusive Sub-License of First-in-class Dissociative Steroid Vamorolon
Pratteln and Allschwil, Switzerland, November 20, 2018 – Santhera Pharmaceuticals (SIX: SANN) and Idorsia Ltd (SIX: IDIA) have entered into an agreement under which Santhera will acquire the option to exclusively in-license, by way of sub-license, the first-in-class dissociative steroid vamorolone in all indications and all countries worldwide except Japan and …
-
14 November
Catabasis and the University of Texas Southwestern Partner to Explore the Potential Benefits of Edasalonexent on Cardiac Function in Duchenne and Becker Muscular Dystrophies
CAMBRIDGE, Mass.–(BUSINESS WIRE)–Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced a collaboration with Pradeep Mammen, MD, FACC, FAHA, founder and Medical Director of the Neuromuscular Cardiomyopathy Clinic at the University of Texas Southwestern (UT Southwestern) Medical Center as well as Co-Director of the National Institute of Health Sponsored UT …
October, 2018
-
8 October
New Data Shows PTC Therapeutics’ Translarna Slows Disease Progression in Children with Duchenne Caused by a Nonsense Mutation
SOUTH PLAINFIELD, N.J., Oct. 6, 2018 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced preliminary data from the first international drug registry for Duchenne patients receiving Translarna™ (ataluren), underscoring the long-term clinical benefit of Translarna when used in routine clinical practice in delaying irreversible muscle loss in children with Duchenne caused by a nonsense …
September, 2018
-
21 September
Catabasis to Present New Edasalonexent Clinical Results and Phase 3 Trial Design in Duchenne Muscular Dystrophy
CAMBRIDGE, Mass.–(BUSINESS WIRE)–Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced that the Company will share three presentations, including in the late breaking session, at the 23rd International Congress of the World Muscle Society, being held October 2 – 6, 2018, at the Intercontinental Hotel in Mendoza, Argentina. Presentations include: Late …
August, 2018
-
30 August
After Phase 2 Failure, Pfizer Terminates Domagrozumab Program for Treatment of Duchenne Muscular Dystrophy
NEW YORK–(BUSINESS WIRE)–Pfizer Inc. (NYSE: PFE) announced today that it is terminating two ongoing clinical studies evaluating domagrozumab (PF-06252616) for the treatment of Duchenne muscular dystrophy (DMD): a Phase 2 safety and efficacy study (B5161002) and an open-label extension study (B5161004). The Phase 2 study (B5161002), did not meet its …
-
16 August
Wave Life Sciences’ WVE-210201 Granted Orphan Drug and Rare Pediatric Disease Designations from the FDA for Treatment of DMD
CAMBRIDGE, Mass., Aug. 16, 2018 (GLOBE NEWSWIRE) — Wave Life Sciences Ltd. (NASDAQ: WVE), a biotechnology company focused on delivering transformational therapies for patients with serious, genetically-defined diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted both orphan drug designation and rare pediatric disease designation for …
June, 2018
-
27 June
Catabasis Presents New Edasalonexent Clinical Biomarker Data Showing NF-kB Inhibition and Target Engagement in DMD
CAMBRIDGE, Mass.–(BUSINESS WIRE)–Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today reported new NF-kB inhibition biomarker results showing edasalonexent target engagement in the MoveDMD Phase 2 trial and open-label extension in boys affected by Duchenne muscular dystrophy (DMD). NF-kB is a fundamental driver of disease progression in DMD. These results add …
-
20 June
Sarepta Announces Positive Preliminary Results from Phase 1/2a Gene Therapy Trial in Duchenne Muscular Dystrophy
CAMBRIDGE, Mass., June 19, 2018 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases, announced that at the Company’s R&D Day, Jerry Mendell, M.D. of Nationwide Children’s Hospital presented positive preliminary results from …
-
19 June
FDA Lifts Clinical Hold on Solid Biosciences’ Clinical Trial for its Investigational Duchenne Muscular Dystrophy Treatment
CAMBRIDGE, Mass., June 18, 2018 (GLOBE NEWSWIRE) — Solid Biosciences Inc. (NASDAQ:SLDB) today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on IGNITE DMD, the Company’s Phase I/II clinical trial for its investigational microdystrophin gene transfer, SGT-001, for the treatment of Duchenne muscular dystrophy (DMD). …