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 ...

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

Children’s Hospital Los Angeles (CHLA) is expanding its safety protocol for patients receiving Elevidys®, the FDA-approved ...

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 ...

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

Chandni Chowk MP Praveen Khandelwal on Tuesday wrote to Prime Minister Narendra Modi, seeking special policy support for the ...

Researchers from Leiden University Medical Center and the University of Florida have found hundreds of blood proteins in ...

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

“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 ...

Santhera Pharmaceuticals has announced positive topline results from a long-term analysis of AGAMREE (vamorolone) in patients ...