Company's Biologics License Application on track with PDUFA target action date of August 22, 2026– SAN DIEGO, June 26, 2026 ...

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 ...

About Elixirgen Therapeutics, Inc. Elixirgen Therapeutics, Inc. is a clinical-stage biotechnology company developing treatments for rare diseases and aging-associated diseases using its ZSCAN4 ...

Deramiocel demonstrates durable skeletal and cardiac muscle benefit over five years in the HOPE-2 OLE studyPUL 2.0 year-over-year decline ...

In human biology, the protein dystrophin is a shining example of Joni Mitchell’s classic line, “you don’t know what you’ve got ’til it’s gone.” Dystrophin stabilizes muscle cells. In its absence, the ...

Panelists discuss how exciting ongoing research efforts are expanding similar gene therapy technologies to other muscular dystrophies like FSHD and myotonic dystrophy, using strategies to knock down ...

Researchers combined advanced imaging techniques and theoretical physics to observe and explain how nanoclusters of the protein emerin form inside living cells. The study uncovers the molecular 'rules ...

A research team led by Associate Professor Hidetoshi Sakurai and Researcher Nana Takenaka-Ninagawa recently demonstrated the superior therapeutic potential of iPS cell-derived mesenchymal stromal ...

Wedbush initiated coverage on Solid Biosciences, Inc. (NASDAQ:SLDB), a gene therapy company focused on treating Duchenne muscular dystrophy (DMD). DMD is a genetic disease that causes progressive ...

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 ...

Duchenne muscular dystrophy (DMD) is a genetic condition that causes increasing muscle weakness over time. While DMD currently has no cure, researchers are studying potential new treatments. When ...

Eight therapies are approved for Duchenne muscular dystrophy (DMD) in the U.S., and all have been approved since 2016. "It took 30 years from the discovery of the gene associated with Duchenne ...