Givinostat is an oral histone deacetylase (HDAC) inhibitor approved by the FDA in 2024 to treat Duchenne patients ages 6 years and older. Pathologic upregulation of HDAC occurs in DMD, leading to ...

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

As of May 2025, RGX-202 displayed favourable safety, with no serious adverse events (SAEs) and adverse events of special ...

Data from the Phase Ia/b study demonstrated that SAT-3247 was well-tolerated and safe with a desirable PK profile.

REGENXBIO Inc. (Nasdaq: RGNX) today announced the completion of enrollment in the AFFINITY DUCHENNE® pivotal trial of RGX-202, an investigational gene therapy for the treatment of Duchenne muscular ...

If cancer is a disease of overabundance, where cells divide without restraint and tumors grow despite the body's best interests, then degenerative diseases are disorders of deprivation.

One of those things is the CureDuchenne clinic in Denton. It provides care to underserved and uninsured people with Duchenne. "I think it's good that we're trying to find the cure," Shep said.

The Swiss pharma is paying $12 billion for Avidity Biosciences and its three late-stage antibody oligonucleotide conjugates.

PepGen shifts focus to PGN-EDODM1 for Myotonic Dystrophy Type 1 after discontinuing PGN-EDO51 for Duchenne Muscular Dystrophy ...

Solid Biosciences ((SLDB)) announced an update on their ongoing clinical study.Elevate Your Investing Strategy: Take advantage of TipRanks Premium at 50% off! Unlock powerful investing tools, ...

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