has granted Fast Track designation to AOC 1044 for the treatment of Duchenne muscular dystrophy (DMD) in people with mutations amenable to exon 44 skipping (DMD44). DMD is a rare genetic condition ...

Entrada has secured the authorisation within the EU Clinical Trial Regulation to begin the Phase I/II trial of ENTR-601-45.

Months after causing safety concerns that led the FDA to hit a related trial with a clinical hold, a phase 2 trial of ...

What Is Duchenne Muscular Dystrophy ... For the small number of DMD patients with a gene mutation that can be treated by skipping exon 45 (a certain section of genetic code), doctors can ...

In Duchenne muscular dystrophy, a DMD mutation leads to a lack of dystrophin ... The treatment is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene.

An abnormality in the DMD gene causes Duchenne muscular dystrophy. The DMD gene produces dystrophin, a protein that maintains the structure of muscle cells. People with DMD have mutations (changes ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, shared the following ...

PepGen ends Duchenne drug program after weak Phase 2 results, pivots to DM1 treatment showing early signs of splicing ...

After months of uncertainty and controversy, the FDA has given approval to Sarepta's Duchenne muscular dystrophy ... a confirmed mutation of the dystrophin gene amenable to exon 51 skipping ...

Entrada Therapeutics can plough on with the development of its Duchenne muscular dystrophy ... investigational exon 44 skipping therapy in adults with a confirmed DMD gene mutation, in the ...

Opens in a new tab or window Eight therapies are approved for Duchenne muscular dystrophy ... of boys with DMD. Exon-Skipping Antisense Oligonucleotides DMD is caused by mutations in the DMD ...

Duchenne muscular dystrophy occurs when that gene fails to make dystrophin. Becker muscular dystrophy occurs when a different mutation in the same gene results in some dystrophin, but it's either ...