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....
treatment News
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 that the Ministry of Health, Labour and Welfare (MHLW) in Japan has granted Orphan Drug designation for zeleciment basivarsen (z-basivarsen), for the treatment of myotonic...
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...
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...
For each 5-mg/dL increase in serum TTR level within 28 days of starting treatment, the relative risk reduction of mortality was up to 31.6% through Month 30, confirming the hypothesis that ever better levels of stabilization achieved by treatment with acoramidis, a near-complete (≥90%) TTR stabilizer, lead to ever better clinical outcomes...
Positive phase I/II data presented at the 2025 International Society on Thrombosis and Haemostasis (ISTH) Congress show NXT007 achieved no bleeds requiring treatment in the highest dose groups in people with haemophilia A1 The NXT007 clinical development programme aims to normalise haemostasis and minimise treatment burden2,3 Three phase III clinical...
Phase 1 trial demonstrates encouraging clinical activity of investigational immunotherapy for relapsed or refractory multiple myeloma Novel investigational dual-antigen-targeting immunotherapy binds to B-cell maturation antigen (BCMA) and GPRC5D on myeloma cells, as well as CD3 on T-cells CHICAGO, Ill. — Johnson & Johnson announced initial Phase 1 results of JNJ-79635322 (JNJ-5322), a novel...
Cambridge, England – An international team has shown that the injection of a type of stem cell into the brains of patients living with progressive multiple sclerosis (MS) is safe, well tolerated and has a long-lasting effect that appears to protect the brain from further damage. The study, led by...