News

October 11, 2019October 11, 2019

Catabasis Joins Jain Foundation to Advance Edasalonexent Development for Dysferlinopathy

News

Catabasis Pharmaceuticals and the Jain Foundation have started a preclinical research collaboration to study edasalonexent as an oral treatment candidate for dysferlinopathy, a group of muscle diseases that includes ... Read more

October 9, 2019October 9, 2019

Gene Therapy SRP-9003 Showing ‘Very Encouraging’ Results at 9 Months in Limb Girdle MD, Sarepta Reports

News

New nine-month data from a Phase 1/2 trial show that a single administration of the gene therapy SRP-9003 (formerly, MYO-101) at low dose significantly improved functional measures and lowered the levels of ... Read more

October 7, 2019October 7, 2019

Request to Approve Viltolarsen, Exon 53 Skipping Treatment for Duchenne MD, Before FDA

News

Nippon Shinyaku filed a request for the approval of viltolarsen (NS-065/NCNP-01) to treat Duchenne muscular dystrophy amenable to exon 53 skipping with the U.S. Food and Drug Administration (FDA), the company announced ... Read more

October 4, 2019October 4, 2019

First Baby Tested for Duchenne in PPMD’s Newborn Screening Pilot Program in NY

News

Parent Project Muscular Dystrophy (PPMD) announced the screening of a first baby for Duchenne muscular dystrophy (DMD) within the organization’s pilot newborn screening program in New York. The $5 million pilot ... Read more

October 2, 2019October 2, 2019

FDA Awards $1M to Phase 2 Study of Ifetroban in Treating Heart Disease Linked to Duchenne MD

News

Cumberland Pharmaceuticals announced that it has been awarded a $1 million grant by the U.S. Food and Drug Administration (FDA) to support a Phase 2 clinical trial into oral ... Read more

September 30, 2019September 30, 2019

CureDuchenne Plans Oct. 11-13 ‘Futures’ Meet at California’s Disneyland

News

About 700 people have registered to attend CureDuchenne’s Futures conference taking place Oct. 11-13 in suburban Los Angeles — more than six times the number of participants at the ... Read more

September 27, 2019September 27, 2019

PPMD Announces First Adult Certified Duchenne Care Center

News

Expanding certification of clinics that provide optimal Duchenne muscular dystrophy care, Parent Project Muscular Dystrophy (PPMD) has announced its first Adult Certified Duchenne Care Center. An extension of the ... Read more

September 27, 2019September 27, 2019

NORD 2019 Rare Disease Summit Set for Oct. 21-22 in Washington, DC

News

Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board ... Read more

September 25, 2019September 28, 2019

Final Tests Planned into Potential Gene Therapy for OPMD, Could Support Trial in Patients

News

Three new animal-model tests of a potential gene therapy for oculopharyngeal muscular dystrophy (OPMD), known as BB-301, are planned, Benitec Biopharma announced. Results are expected to support a request for a Phase ... Read more

September 23, 2019October 2, 2019

CureDuchenne Offers One-Stop Informative Guide on Durable Medical Equipment

News

To help families find the ideal equipment at every stage of need for those with Duchenne muscular dystrophy (DMD), CureDuchenne launched on its website a comprehensive and interactive guide. Presenting ... Read more

Pin It on Pinterest