Marathon Pharmaceuticals, a company that develops new medications for rare diseases with a focus on patients who have no treatment options, recently…
Articles by Margarida Azevedo, MSc
Researchers at the University of Michigan Department of Molecular & Integrative Physiology have deciphered a molecular connection leading to a potential…
Researchers at the University of Missouri have reported the complete and successful treatment of Duchenne muscular dystrophy (DMD) in dogs…
Parent Project Muscular Dystrophy (PPMD) announced the submission of results from its recent patient-centered benefit-risk assessment (PCBR) study to the…
A heart rate-corrected 6-minute walk test (6MWT) is a more accurate outcome measure than the standard 6MWT, and should be…
Developer of novel RNA-targeted therapeutics, Sarepta Therapeutics, recently announced the U.S. Food and Drug Administration’s (FDA) Peripheral and Central Nervous System…
PTC Therapeutics, Inc., reported last week its findings from a Phase III, double-blind, placebo-controlled, 48-week ACT study of Translarna (ataluren) in…
In a recent study published in the journal PLOS Medicine, a team of researchers from the University…
New research has shown that a RNA technique called “exon skipping” can successfully generate stable proteins and slow down a severe disease…
Alorica, an Irvine, California-based, company, recently announced it will join forces with non-profit health organization Muscular Dystrophy Association (MDA) to help fundraise for…