WALTHAM, Mass. — Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage company focused on delivering functional improvement for people living with genetically driven neuromuscular diseases, today announced positive topline results from the Registrational Expansion Cohort (REC) of its Phase 1/2 DELIVER trial evaluating zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251) in...
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– Recently presented data demonstrated sustained functional improvement with DYNE-251 treatment through 18 months – – Data from the fully enrolled DELIVER registrational expansion cohort is planned for late 2025 – WALTHAM, Mass. — Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage company focused on advancing life-transforming therapeutics for people living...
Waltham, Mass. — Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today announced that DYNE-251, an investigational therapeutic for Duchenne muscular dystrophy (DMD) mutations amenable to exon 51 skipping, was granted U.S. Food and Drug...
WALTHAM, Mass. — Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage company, today announced that the Ministry of Health, Labour and Welfare (MHLW) in Japan has granted Orphan Drug designation for DYNE-251 in individuals with Duchenne muscular dystrophy (DMD) who have mutations in the DMD gene that are amenable to exon 51 skipping....
Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, reported financial results for the fourth quarter and full year 2022 and business highlights.
WALTHAM, Mass. — Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage company focused on delivering functional improvement for people living with genetically driven neuromuscular diseases, today announced additional one-year data from its ongoing Phase 1/2 ACHIEVE clinical trial of zeleciment basivarsen (z-basivarsen, formerly known as DYNE-101), in patients with myotonic dystrophy...
Dystonia sounds more like an Eastern European country than a health disorder. And like so many other diseases, conditions and disorders, the average person has probably never heard of it. It is only when dystonia affects a friend or family member that people start to ask questions. What is dystonia?...
WOODCLIFF LAKE, N.J. – Eagle Pharmaceuticals, Inc. (“Eagle” or the “Company”) (NASDAQ: EGRX) today announced that TREAKISYM ready-to-dilute (“RTD”) (bendamustine hydrochloride 120 mg/m2) liquid formulation has been approved for a new indication in combination with rituximab (“BR therapy”) as treatment for relapsed or refractory diffuse large B-cell lymphoma (“r/r DLBCL”)...
ORLANDO, Fla. — Johnson & Johnson (NYSE: JNJ) announced today updated results from the Phase 3 CARTITUDE-4 study supporting durable treatment-free remissions as early as second line treatment with CARVYKTI® (ciltacabtagene autoleucel; cilta-cel). In 80 percent of as-treated patients with relapsed or refractory multiple myeloma (RRMM) and standard-risk cytogenetics who were treated with CARVYKTI® as...
RESEARCH TRIANGLE PARK, N.C. – BioCryst Pharmaceuticals, Inc. today announced that the French National Agency for Medicines and Health Products Safety (ANSM) has granted an Autorisation Temporaire d’Utilisation de cohorte (cohort ATU), or Temporary Authorization for Use, for the use of berotralstat to prevent attacks of hereditary angioedema (HAE) in...