Reduced progression of myocardial fibrosis measured by LGE on cardiac MRI (p=0.022)Significant improvement in LVEF versus placebo in patients ...

Tenaya’s Highly Selective HDAC6 Inhibitor TN-301 Outperformed Approved Pan-HDAC Inhibitor Givinostat in Improving Muscle Function and Correcting ...

Genethon, a pioneer and leader in gene therapy for rare diseases, unveiled results at the MDA Conference in Orlando confirming the long-term efficacy of its GNT0004 gene therapy in Duchenne muscular ...

Precision BioSciences Inc. (NASDAQ:DTIL) presented new preclinical findings for its PBGENE-DMD gene therapy at the Muscular ...

PBGENE-DMD, a novel first-in-class gene editing therapy, utilizes a gene excision approach that is clearly differentiated from existing microdystrophin and exon skipping treatments. PBGENE-DMD is ...

The FDA has resumed review of Capricor Therapeutics’ previously rejected Duchenne muscular dystrophy cell therapy following the biotech’s submission of more clinical data, as well as the newly ...

Slowing the loss of ambulation in patients with Duchenne muscular dystrophy (DMD) may also mitigate worsening disease burden and overall function, according to a pair of posters presented at ISPOR ...

"Fast Track designation is an important regulatory milestone for PBGENE-DMD and reflects the significant unmet need in DMD," said Michael Amoroso, Chief Executive Officer of Precision BioSciences. "We ...

"This can't go on," said Sen. Ron Johnson ...

NASHVILLE, Tenn., Feb. 4, 2025 /PRNewswire/ -- Cumberland Pharmaceuticals Inc. (Nasdaq: CPIX), a specialty pharmaceutical company with development efforts focused on new products for rare diseases, ...

Sen. Roger Wicker urges states to add Duchenne muscular dystrophy to newborn screening, after federal health officials back ...

The degree to which novel value elements such as insurance value impact estimated treatment value for rare, severe genetic diseases such as Duchenne muscular dystrophy is unclear. Objectives: To ...