Category Archives: Muscular Dystrophy Treatment

- If approved, KALYDECO (ivacaftor) will be the first and only medicine in Europe to treat the underlying cause of cystic fibrosis in patients with the R117H mutation, the most common residual function mutation, as young as 6 months of age - Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Medicines Agencys (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for the label extension of KALYDECO (ivacaftor), to include the treatment of children and adolescents with cystic fibrosis (CF), ages 6 months and older weighing at least 5 kg who have the R117H mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Continue reading →

(ANSA) - Rome, April 29 - The European Medicines Agency (EMA) has sounded the alarm about the use of cell-based therapies that are promoted as being miracle cures but, in fact, are often unproven and unauthorized. Serious Risks. Continue reading →

BOSTON & WALTHAM, Mass.--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq:VRTX) and Affinia Therapeutics announced today that the two companies have entered into a strategic research collaboration to engineer novel adeno-associated virus (AAV) capsids to deliver transformative genetic therapies to people with serious diseases. Affinia Therapeutics proprietary AAVSmartLibrary and associated technology provides capsids for improved tissue tropism, manufacturability and pre-existing immunity Continue reading →

Dyne Therapeutics, a biotechnology company pioneering targeted therapies for patients with serious muscle diseases, today announced an equity investment from CureDuchenne Ventures. Continue reading →

NASHVILLE, Tenn. (Tuesday, March 31, 2020) Cumberland Pharmaceuticals Inc. (NASDAQ: CPIX) a specialty pharmaceutical company, today announced a national initiative to support hospitals and clinics that use Caldolor (ibuprofen) injection for the treatment of patients with fever, severe hyperthermia and other symptoms associated with COVID-19 infections. Continue reading →

(Photo by Rick Mellerup) Anne Markel-Crozier, a certified social worker, and Matthew Brasette, an attorney, addressed the crowd at a recent lecture at Stockton University in Manahawkin. Continue reading →

Conte G, Gioja L. Scrofola del sistema muscolare Continue reading →

New Jersey, United States:The Duchenne Muscular Dystrophy Treatment Market is carefully researched in the report while largely concentrating on top players and their business tactics, geographical expansion, market segments, competitive landscape, manufacturing, and pricing and cost structures. Each section of the research study is specially prepared to explore key aspects of the Duchenne Muscular Dystrophy Treatment market Continue reading →

Like many young children, summer camp, from water tubing to zip lining to water sliding, represents Ethan LyBrands favorite time of year. It is where the 10-year-old Decatur boy, despite his battle with Duchenne Muscular Dystrophy and dependence on a power chair, can just be a kid Continue reading →

EDITOR'S NOTE: Please note that due to the ongoing COVID-19 pandemic, some of the following meetings/events may be cancelled or rescheduled unexpectedly. The Purple Heart Chapter of Sumter is sponsoring a project to have all Purple Heart medal recipients inducted into the National Purple Heart Hall of Honor located in New York. Continue reading →