"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 ...

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 ...

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 ...

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 ...

“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 ...

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 ...

BOULDER, Colo.--(BUSINESS WIRE)--Edgewise Therapeutics, Inc. (NASDAQ: EWTX), a clinical-stage biopharmaceutical company focused on developing orally bioavailable, small molecule therapies for the ...

Born with muscular dystrophy, she received a MacArthur “Genius” grant in 2024 for her decades of calling attention to the ...

Hirano at Columbia University, an MDA Care Center, to develop a TK2-deficient mouse model, which proved that targeted treatments could restore mitochondrial function. This led to FDA granting ...

BOULDER, Colo.--(BUSINESS WIRE)--Edgewise Therapeutics, Inc., (NASDAQ: EWTX), a clinical-stage biopharmaceutical company focused on developing orally bioavailable, small molecule therapies for the ...