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 ...

Some children in Northern Ireland who live with a severe form of muscular dystrophy will be able to access new medication on the health service, BBC News understands. The move from the Belfast Health ...

WASHINGTON, June 12, 2025 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), is pleased to announce the ...

His parents Stephen and Jenny have asked Sheffield Children's NHS Foundation Trust to approve the use of drug givinostat, ...

Givinostat is an oral histone deacetylase (HDAC) inhibitor approved by the FDA in 2024 to treat Duchenne patients ages 6 ...

Please provide your email address to receive an email when new articles are posted on . The FDA has approved an abbreviated new drug application for a generic version ...

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

From Corpus Christie, Texas, Jenn Mirabal, an MDA staff member and mother of a child living with Duchenne muscular dystrophy ...

Duchenne muscular dystrophy (DMD) results from changes in the DMD gene. Children may inherit DMD gene changes from their parents or have spontaneous gene changes. The DMD gene tells the body how to ...

Duchenne muscular dystrophy (DMD) is an inherited muscle disorder that causes progressive breakdown of muscle tissue. Symptoms typically begin in early childhood, and most parents and caregivers ...

If a boy in New York has been convinced to think he’s a girl, the state Medicaid program has got you covered. But if a boy ...

Each week, this series shares MarionMade! stories of our many wonderful people, places, products and programs in the greater Marion community. To read more positive stories of Marion, or to share some ...