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 ...

Catalyst is offering patients and their caregivers a personalized program called Catalyst Pathways to provide guidance and financial support. Agamree ® (vamorolone) oral suspension has been made ...

Experts discuss the landscape of treatment options for Duchenne muscular dystrophy, examine the benefits and risks of gene therapy and conventional steroid treatments, and emphasize the need for early ...

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

“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 ...

Benitec Biopharma Inc. (NASDAQ: BNTC) (“Benitec” or the “Company”), a clinical-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on its proprietary “Silence ...

SAN DIEGO, June 25, 2024 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare ...

Share on Facebook. Opens in a new tab or window Share on Bluesky. Opens in a new tab or window Share on X. Opens in a new tab or window Share on LinkedIn. Opens in a new tab or window Eight therapies ...

BridgeBio said an experimental therapy for a rare form of muscular dystrophy succeeded in a key trial, potentially setting up ...