STRASBOURG, France and PHILADELPHIA – Dynacure, a clinical-stage company focused on developing and commercializing novel therapies to transform the lives of patients with rare diseases who have limited or no treatment options, today announced its recognition of Rare Disease Day 2021. Dynacure is helping to raise awareness of myotubular and...
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WALTHAM, Mass. — Dyne Therapeutics, Inc., a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today announced positive initial clinical data from its ACHIEVE trial of DYNE-101 in patients with myotonic dystrophy type 1 (DM1) and its DELIVER trial of DYNE-251...
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...
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.
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”)...
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...
New research presented this week at ACR Convergence, the American College of Rheumatology’s annual meeting, found that patients started on early, aggressive treatment with a combination of biologic and conventional disease modifying antirheumatic drugs (DMARDs) achieved clinically inactive disease in children with polyarticular juvenile idiopathic arthritis (JIA) more frequently compared...
OAK BROOK, Ill. – A new study found increased coronary vessel wall thickness that was significantly associated with impaired diastolic function in asymptomatic, middle-aged individuals living with HIV. The study was published today in Radiology: Cardiothoracic Imaging, a journal of the Radiological Society of North America (RSNA). According to the...
Boca Raton, Florida – Endometriosis is a common, burdensome, chronic disease that affects more than 11% of women of reproductive age in the United States and 190 million women worldwide. Early diagnosis remains a major clinical and public health challenge. The average time to diagnose endometriosis is seven years after...