FDA Approves KYGEVVI® (doxecitine and doxribtimine), the First and Only Treatment for Adults and Children Living with Thymidine Kinase 2 deficiency (TK2d), a Rare and Devastating Mitochondrial Disease ...

“The approval today of KYGEVVI® (doxecitine and doxribtimine), provides real hope for patients and families living with this devastating disease,” said Angela Lek, PhD, interim Chief Research Officer ...

"In many countries, health insurance changes when DMD men are no longer seen in a specialized pediatric center. This impacts massively on the quality of care they receive. For this reason, many DMD ...

ST. LOUIS — On a beautiful day in Forest Park, dozens of people brought hope to kids and adults with Muscular Dystrophy by just taking a long walk. The Muscular Dystrophy Association used the walk to ...

The University at Albany and Virginia Commonwealth University were awarded a $6.5 million NIH grant to establish a federal ...

Editor’s note: This is an automatically generated transcript. Please notify [email protected] if there are concerns regarding accuracy of the transcription. Patient advocacy organizations often bring ...

Adults with Duchenne muscular dystrophy demonstrated functional improvements exceeding natural history, following 28-day treatment with SAT-3247 Grip strength increased 118.6% and predicted forced ...

Edgewise Therapeutics Inc EWTX released 12-month topline results from the ARCH study, an open-label, single-center study assessing EDG-5506 in adults with Becker Muscular Dystrophy (BMD). EDG-5506 is ...

Company now has three distinct rare disease programs in clinical development - myotonic dystrophy type 1 (DM1), facioscapulohumeral muscular dystrophy (FSHD), and Duchenne muscular dystrophy (DMD) ...

BOULDER, Colo.--(BUSINESS WIRE)--Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the Company will present on EDG-5506, an orally ...

The muscular dystrophies are commonly associated with cardiovascular complications, including cardiomyopathy and cardiac arrhythmias. These complications are caused by intrinsic defects in ...