DMD

Oct 29, 2024

FDA Grants Orphan Drug Status to MDL-101 for LAMA2-CMD Treatment Modalis Therapeutics Corporation has received Orphan Drug Designation (ODD) from the FDA for MDL-101, its promising treatment for congenital muscular dystrophy type 1A (LAMA2-CMD). This designation is a significant step, aimed at fostering the deve...

Aug 23, 2024

Duchenne Muscular Dystrophy (DMD) is a rare disorder, but it is one of the most common genetic conditions, affecting roughly 1 in every 3,500 male births worldwide. Gene therapy for Duchenne Muscular Dystrophy (DMD) is poised to revolutionize the treatment landscape by addressing the underlying genetic cause of the...

Aug 20, 2024

Sarepta Therapeutics, a leader in precision genetic medicine for rare diseases, has recently achieved a significant milestone with the FDA's approval of AMONDYS 45 (casimersen). This antisense oligonucleotide, developed from Sarepta’s phosphorodiamidate morpholino oligomer (PMO) platform, is now approved for treati...

Aug 20, 2024

Duchenne muscular dystrophy (DMD) is a rare genetic disorder usually diagnosed in young boys, gradually weakening muscles across the body until the heart or lungs fail. Symptoms often show up by the age of 5; as the disease progresses, patients tend to lose the ability to walk around the age of 12. As per a recent ...

Jun 30, 2023

Duchenne muscular dystrophy (DMD) is a rare neuromuscular disease that affects 1 in every 3,500 to 5,000 male neonates worldwide. It is caused by mutations in the gene encoding the protein dystrophin. These genetic changes emerge as developmental delays and, in more severe types of DMD, limb weakness, loss of indep...

Sep 11, 2018

Nucala of GlaxoSmithKline exhibits splendid benefits to other IL-5 Inhibitors in Asthma, however flunks as a COPD Drug A Complete Response Letter is received by GSK from the U.S. Food and Drug Administration (FDA) for Nucala (mepolizumab) as COPD treatment. Nucala, which targets the IL-5 pathway, was initially appro...