Margarida Azevedo, MSc,  —

The effects of glucocorticoid therapy and bisphosphonate treatment on tissue structure and bone matrix mineralization of nine boys with Duchenne Muscular Dystrophy (DMD) was recently highlighted in a research paper entitled “Histomorphometry and Bone Matrix Mineralization Before and After Bisphosphonate Treatment in Boys with Duchenne Muscular Dystrophy: A Paired Trans-Iliac…

Marathon Pharmaceuticals has announced the launch of ACCESS DMD, its expanded access program (EAP) to provide deflazacort to qualified patients with Duchenne muscular dystrophy (DMD) under the authorization of the U.S. Food and Drug Administration (FDA). Marathon Pharmaceuticals, LLC, a biopharmaceutical company that develops treatments for rare neurological diseases,…

Results from a recent study published in the Journal of Pedriatric Orthopaedics showed full-time wheelchair use is a risk factor of first fracture among pediatric male patients with Duchenne or Becker muscular dystrophy. However, no correlation was found between fracture and corticosteroid use in these patient populations. Muscular…

Results from a study observing myotonic dystrophy type 2 (DM2) patients has helped pinpoint which disease symptoms are most important to patients with this rare type of muscular dystrophy. According to the study authors, the findings could help create a roadmap for doctors to prioritize treatments. The study titled “Patient-Reported…

Ringo, a golden retriever, has made an important contribution to science by helping researchers discover a gene that inhibits the consequences of dystrophin loss in Duchenne muscular dystrophy (DMD). Even though Ringo carried the classic DMD mutation, he and one of his male offsprings had a variant in the Jagged1 gene that allowed…

aTyr Pharma, Inc., has announced an expansion of its Phase 1b/2 clinical trial to continue the development and evaluation of efficacy and safety of the Resolaris clinical program, for the treatment of early onset facioscapulohumeral muscular dystrophy (FSHD) patients. Facioscapulohumeral muscular dystrophy (FSHD) is a genetic muscular disorder, characterized by progressive muscle…

ISIS Pharmaceuticals, a company focusing on the development of RNA-targeted drug discovery using its antisense technology platform, announced it earned a $2.8 million milestone payment from Biogen to continue developing the ongoing Phase 1/2a study of ISIS-DMPK-2.5Rx for the treatment of myotonic dystrophy type I (DM1) patients. This payment stems from a collaboration with Biogen and…

Ironwood Pharmaceuticals, Inc., recently announced the beginning of a Phase Ib clinical trial of IW-1973 and a Phase Ia clinical trial of IW-1701, both soluble guanylate cyclase (sGC) stimulators from a wide-ranging, pharmacologically distinct portfolio of sGC composites discovered by the company. The clinical results intend to determine dose selections along with the main…

Recent research has demonstrated that early bisphosphonate treatment of a mice model of Duchenne muscular dystrophy (DMD) had some positive effects on bone integrity and skeletal muscle strength. The research paper, entitled “Positive effects of bisphosphonates on bone and muscle in a mouse model of Duchenne muscular dystrophy,” was…

Professor Liza Pon, PhD, a leading researcher at the department of pathology and cell biology at Columbia University in New York, was recently awarded a $300,000, three-year research grant from the Muscular Dystrophy Association (MDA) to continue her work in congenital muscular dystrophy (CMD). In a recent interview conducted by…