Wave Life Sciences, Ltd., is planning to begin clinical testing of its next-generation nucleic acid therapy for patients…
Articles by Margarida Azevedo, MSc
Santhera Pharmaceuticals announced updates on regulatory filings for Raxone (idebenone), saying it plans to submit comprehensive briefing material and a meeting request…
Lowe’s, a nationwide home improvement company, announced it has raised over $7.5 million across its more than 25,000 U.S. retail stores…
Parent Project Muscular Dystrophy (PPMD) is reaching out to families affected by Duchenne muscular dystrophy (DMD) through its ‘Every Single…
Parent Project Muscular Dystrophy (PPMD), the largest U.S. nonprofit group focused on Duchenne muscular dystrophy (DMD), is holding a Pulmonary Outcomes Workshop this…
The Muscular Dystrophy Coordinating Committee (MDCC) has released an updated version of its Action Plan for the Muscular Dystrophies,…
Researchers at Switzerland’s Basel University Hospital investigated the biochemical and physiological properties of the muscles involved in the control of the…
Marathon Pharmaceuticals recently presented findings from a Phase 3 trial comparing the effects of deflazacort to prednisone and placebo in boys with Duchenne muscular…
Marathon Pharmaceuticals has announced its investigational medicine deflazacort will be accessible for free through an Expanded Access Program (ACCESS…
Ultragenyx, a biopharma dedicated to the development of investigational products for rare diseases, will fund the efforts of a…