Isaura Santos,  —

Catabasis, a Cambridge biotech startup, is currently advancing a platform to simultaneously modulate multiple targets in disease pathways. Researchers there are especially focused on diseases like Duchenne muscular dystrophy (DMD) and Amyotrophic lateral sclerosis (ALS). The startup is now in the middle of a fundraise, striving to reach $20.4 million, according…

The Muscular Dystrophy Association (MDA) recently announced that MDA clinics are offering genetic testing for those with limb-girdle muscular dystrophy (LGMD) thanks to a grant from Sanofi company Genzyme in collaboration with Emory Genetics Laboratory. This represents a major step in diagnostic advances that will help those with the disease and their respective clinicians so they can find…

PhaseBio Pharmaceuticals, Inc., a leading company in biopolymer-based drugs developing treatments to address metabolic and specific cardiopulmonary disorders, recently announced that a $40 million financing round led by AstraZeneca has been closed. This investment from AstraZeneca, Hatteras Venture Partners, New Enterprise Associates and Johnson & Johnson Innovation – JJDC and Fletcher Spaght Ventures will serve…

Santhera Pharmaceuticals‘ Principal Investigator Gunnar M. Buyse will be presenting Phase III data on Idebenone (Raxone®/Catena®) for the treatment of Duchenne Muscular Dystrophy (DMD) at the Annual Meeting of the American Academy of Neurology (AAN). Buyse’s oral presentation entitled, “Idebenone Reduces Loss of Respiratory Function in Duchenne Muscular Dystrophy-Outcome…

The Stanford Comprehensive Neuromuscular Clinic at Lucile Packard Children’s Hospital (Stanford) was recently named by the Parent Project Muscular Dystrophy (PPMD) as a Certified Duchenne Care Center. The PPMD is a nonprofit organization on the forefront of the fight against Duchenne…

The FSH Society, a charity organization based in Massachusetts supporting research and advocacy related to facioscapulohumeral muscular dystrophy (FSHD) has recently announced the launch of the #CureFSHD campaign. The aim of this campaign is to boost awareness and to educate people about FSHD, which is a type of…

Charles Gersbach, Muscular Dystrophy Association research grantee and Biomedical Engineering Professor at Duke University, has recently announced an enormous and potentially game-changing development in gene modification for young men and boys suffering from Duchenne muscular dystrophy (DMD). The results were published in Nature Communications in a…

BioBlast Pharma, a clinical-stage biotechnology firm focused on developing orphan disease drugs, recently announced it has received an allowance from the United States Patent and Trademark Office (USPTO) regarding a method to address oculopharyngeal muscular dystrophy (OPMD) with Cabaletta.

ReveraGen BioPharma recently announced the start of a Phase 1 clinical trial of a new disassociate steroidal drug called VBP15, indicated to address Duchenne muscular dystrophy (DMD). Recruitment for the trial is currently underway, and the initial doses have already been administered in the first volunteers. “Glucocorticoids are a…

Researchers working in public health have recently published a report based on the first broad study in the United States on the frequency of two common muscle-weakness disorders that mostly affect boys: Duchenne muscular dystrophy and Becker muscular dystrophy. The study was published in …