Research

Catabasis is enrolling boys ages 4 to 7 (up to 8th birthday), any mutation type, who have not been on steroids for at least the past six months.   What is Edasalonexent? Edasalonexent [...]

Sarepta Therapeutics, Inc., a leader in precision genetic medicine for rare diseases, announced positive results from its interim analysis of muscle biopsy endpoints comparing casimersen [...]

February 25, 2019 Santhera Pharmaceuticals announces results from the SYROS study.   The primary objective of this study was to evaluate the long-term evolution of the respiratory function [...]

The success of CRISPR in a model of Duchenne muscular dystrophy Original story from Duke University Researchers at Duke University have shown that by using genome editing technology, CRISPR can [...]

PTC124-GD-041-DMD (Also known as study 041) The clinical trial of ataluren in nonsense mutation Duchenne muscular dystrophy (NMDMD) Study 041 is a new clinical trial for boys and young men who [...]

Sarepta Announces FDA Acceptance of Golodirsen (SRP-4053) New Drug Application for Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 53 Press release here: Sarepta.com   [...]

Sarepta Therapeutics completes submission of New Drug Application seeking approval of golodirsen in patients with DMD amenable to skipping exon 53 Press Release here Golodirsen has been studied [...]

One of the objectives of our team is to inform you about new treatments. Here’s the latest news about Polaris DMD, the clinical trial of edasalonexent, developed by Catabasis. Thank you for [...]

Here’s the latest news from the University of Buffalo (UB) about an experimental therapy called GsMTx4. Based on a molecule found in tarantula venom, this “protein” can prevent muscle mass loss [...]

One of the objectives of our team is to inform you about new treatments. Here’s the latest news from Sarepta Therapeutics. They announce that at the 23rd International Congress of the World [...]