It is debatable, however, whether this creates a problem for society, because even though the frequency of the lethal mutations would increase, the number of babies born with DMD would decrease.

Entrada Therapeutics has secured authorisation from the UK's Medicines and Healthcare Products Regulatory Agency (MHRA) to ...

While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several ...

Cumberland Pharmaceuticals Inc. (NASDAQ:CPIX) released topline results from its Phase 2 FIGHT DMD trial. The study evaluated ...

Duchenne muscular dystrophy results from mutations in the DMD gene that encodes the dystrophin protein. There are many types of mutations that can cause the disease; each disrupts the reading frame ...

Cumberland Pharmaceuticals Inc. (Nasdaq: CPIX), a specialty pharmaceutical company with development efforts focused on new products for rare diseases, today announced positive top-line results from ...

Around 13% of DMD patients respond to exon 51 skipping, but PepGen reckons other drugs in its pipeline will address mutations that are central to around 35% of all cases of the disease.

Swiss biotech Santhera Pharmaceuticals has started launch preparations for its Duchenne muscular dystrophy (DMD) drug Agamree (vamorolone) in the UK, following a recommendation from England’s ...

The exon-skipping drug was approved by the FDA for DMD patients with mutations in the dystrophin gene amenable to exon 53 skipping, a group that accounts for around 8% of the total DMD population.

Entrada plans to initiate clinical testing of ENTR-601-44 in Duchenne MD patients amenable to exon 44 skipping later this year.

Wave Life Sciences develops stereopure oligonucleotides targeting genetic mutations, with lead assets in DMD, AATD, and Huntington's Disease showing promising data. Major collaborations with GSK ...