News

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…

“Can some of them get better, Daddy?” a young boy asks his father on the screen. By “them,” he means people with Duchenne muscular dystrophy (DMD). The boy, Theodor Taussig, who also has DMD, wants to believe he is getting better, and hopes as much as one can that…

At its first virtual investor event, biotech company Centogene set a bold mission: to cure 100 rare diseases within the next decade. A leader in the field of genetic diagnostics, Centogene used the June 22 event to present its strategic priorities, outlining its plans to speed the discovery…

Oral losmapimod, an investigational treatment for people with facioscapulohumeral muscular dystrophy (FSHD), showed clinically relevant benefits over a placebo on multiple measures of disease progression and patient-reported outcomes after almost a year, according to data from the ReDUX4 Phase 2b clinical trial. But the therapy failed to reach…

Vita Therapeutics announced that it has raised $32 million in financing to advance its work into cell-based therapies for muscular dystrophy. “At Vita Therapeutics our mission is to deliver long-term disease-modifying cell engineered treatments for patients living with muscular dystrophies and other high unmet medical needs,” Douglas Falk, the…

CureDuchenne has launched the first comprehensive data hub to connect biosamples and clinical and genetic data from people with Duchenne and Becker muscular dystrophies — as well as disease carriers — with clinicians, researchers, and therapy developers. Called CureDuchenne Link, this next-generation data-sharing platform is expected…

To help patient advocacy leaders and their partners better understand how global health statistics codes — known as ICD codes — are assigned, updated, and revised in the U.S. health information system, the EveryLife Foundation for Rare Diseases is presenting a first-of-its-kind resource guide. The foundation created the…

Those who wish to gain practical tools for living optimally with rare diseases are encouraged to attend the annual Living Rare Living Stronger Patient and Family Forum, hosted by the National Organization for Rare Disorders (NORD) and set this year for June 26-27. The conference brings together patients,…