CureDuchenne has teamed up with Brigham and Women’s Hospital, in Boston, to launch the nation’s first free newborn screening initiative for Duchenne muscular dystrophy (DMD). The partnership establishes Brigham and Women’s as the only U.S. birth hospital to offer parents the choice of screening their new baby for…
After being diagnosed as a carrier of x-linked myotubular myopathy (XLMTM) and losing her infant twin boys to the disease, Ashley Walker was driven into an episode of alcoholism. What she didn’t know then was that her liver was affected severely by the disease, which eventually resulted in an…
A treatment combination of two blood pressure medicines can help slow heart function decline in males with Duchenne muscular dystrophy (DMD), a single center study suggests. These findings support international guidelines that recommend the use of cardiac medications to help stabilize heart function in DMD. The study, “…
AllianceRx Walgreens Prime, a specialty and home delivery pharmacy, is partnering with TailorMed, a healthcare technology company, to help lower out-of-pocket prescription costs for specialty pharmacy patients. Medications attained through specialty pharmacies are those used to treat rare and chronic conditions in the U.S., and are often extremely costly. For…
A Phase 1b clinical trial evaluating Astellas Pharma’s ASP0367 is recruiting boys with Duchenne muscular dystrophy (DMD), ages 8–16, at six U.S. sites. The ongoing trial (NCT04184882) is evaluating the safety and tolerability of the investigational oral therapy, which is designed to improve muscle function in patients with DMD,…
AMRA Medical’s new MRI method was found to play an instrumental role in detecting changes in disease progression among individuals with facioscapulohumeral muscular dystrophy (FSHD) in a recently completed clinical trial, according to the company. The new MRI technique, introduced in the Phase 2b ReDUX4 study, provides…
Nominations are now open for the worldwide 2022 Black Pearl Awards from Eurordis-Rare Diseases Europe. The 12 award categories recognize individual advocates, policy makers, researchers, organizations, and companies who work to make a difference for the global rare disease community. The deadline for nominations is Sept. 10…
Long-term treatment with Viltepso (viltolarsen) safely and effectively prevents a decline in motor function in boys with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping. Those are the findings of two-year data from a Phase 2 trial of Viltepso, an exon-skipping therapy being developed by…
Givinostat halted muscle shrinkage and fat infiltration in men with Becker muscular dystrophy (BMD) despite failing to ease muscle scarring, compared to a placebo, according to top-line, one-year results of an ongoing Phase 2 clinical trial. “We are very encouraged by the significant difference in muscle fat infiltration…
The U.S. Food and Drug Administration (FDA) has granted fast track designation to LION-101, an investigational gene therapy that Asklepios BioPharmaceutical (AskBio) is developing as a treatment for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). This designation is given to therapies designed to fill an unmet need in medical care for…
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