Muscular Dystrophy News

January 13, 2022January 13, 2022

Phase 1/2 Trial Set to Test RGX-202, Gene Therapy for Duchenne MD

The U.S. Food and Drug Administration (FDA) has approved Regenxbio’s request to launch a Phase 1/2 clinical trial in the U.S. evaluating the safety and efficacy of RGX-202, its experimental ... Read more

January 11, 2022January 11, 2022

SRP-9001 Benefits Duchenne Patients Originally Given Placebo in Trial

Muscular Dystrophy News

Children with Duchenne muscular dystrophy (DMD) who had been given a placebo in a clinical trial experienced a marked improvement in motor abilities after being treated with Sarepta Therapeutics‘ experimental ... Read more

January 6, 2022January 7, 2022

EDG-5506 Reduces Key Biomarkers of Muscle Damage in Early BMD Trial

Muscular Dystrophy News

The experimental oral therapy EDG-5506 is generally safe and results in rapid and profound reductions in the levels of muscle damage biomarkers in men with Becker muscular dystrophy (BMD), top-line data from ... Read more

January 4, 2022January 5, 2022

CureDuchenne Invests $1M in Gennao Bio’s Effort to Find Therapies

Muscular Dystrophy News

Genetic medicines company Gennao Bio will use a $1 million CureDuchenne Ventures investment to help expand its gene monoclonal antibody (GMAB) platform technology, which is now applied to rare muscle ... Read more

December 28, 2021December 28, 2021

Father-daughter Duo Double Up to Find FSHD, Rare Disease Cures

Muscular Dystrophy News

When his daughter, Meredith Huml, was diagnosed — finally — with facioscapulohumeral (FSHD) muscular dystrophy in 2004, Raymond Huml immediately jumped into action, researching everything he could about the disease ... Read more

December 23, 2021December 23, 2021

Pfizer Reports Death of Participant in DMD Gene Therapy Trial

Muscular Dystrophy News

A young man with Duchenne muscular dystrophy (DMD), who was participating in Pfizer’s Phase 1B open-label clinical trial evaluating its gene therapy candidate PF-06939926, has died. The patient has only ... Read more

December 21, 2021December 22, 2021

Plans Advancing for Trial of Elamipretide in Aiding Cardiac Muscle in DMD

Muscular Dystrophy News

A branch of the U.S. Food and Drug Administration (FDA) known as the Division of Cardiology and Nephrology has given positive feedback to Stealth BioTherapeutics‘ program for Duchenne muscular dystrophy (DMD). In ... Read more

December 16, 2021December 16, 2021

Elamipretide as Add-on May Boost Efficacy of Exon-skipping Therapies

Muscular Dystrophy News

Elamipretide, an investigational therapy designed to increase the activity of mitochondria, may boost the effectiveness of exon-skipping therapies for Duchenne muscular dystrophy (DMD), its developer, Stealth BioTherapeutics, reported. “Our new preclinical ... Read more

December 14, 2021December 15, 2021

UK Panel Expands Care Guidelines for Adults With DMD

Muscular Dystrophy News

The U.K. Adult North Star Network (ANSN) has developed a set of guidelines for best care management of adults with Duchenne muscular dystrophy (DMD) based on consensus among experts in ... Read more

December 9, 2021December 9, 2021

Atamyo Cleared in UK to Test ATA-100 for Limb-Girdle MD Type R9

Muscular Dystrophy News

Atamyo Therapeutics has been given the go-ahead to start clinical testing of ATA-100, the company’s investigational gene therapy for a specific form of limb-girdle muscular dystrophy (LGMD) called type R9 (LGMDR9). ... Read more

December 7, 2021December 7, 2021

‘Ordinary Joe’ Actor With MD Brings Life to TV Character With Disability

Muscular Dystrophy News

The show’s name may be “Ordinary Joe,” but this NBC drama series is proving extraordinary for its authentic depiction of a character with a disability, portrayed by John Gluck, 13, ... Read more

December 2, 2021December 3, 2021

Vamorolone Shows Efficacy With Fewer Side Effects Over 48 Weeks

Muscular Dystrophy News

Treatment with vamorolone continued to show a good safety profile and sustained efficacy across functional assessments after almost one year in boys with Duchenne muscular dystrophy (DMD), according to 48-week ... Read more

November 30, 2021November 30, 2021

Elijah Stacy Shares 13 Life Lessons in Book About Experience With DMD

COVID-19 News, Muscular Dystrophy News

Author and advocate Elijah J. Stacy is using what he has learned through adversity in battling Duchenne muscular dystrophy (DMD) to help others overcome their own challenges. DMD is a ... Read more

November 23, 2021November 23, 2021

DMD Gene Therapy Candidate RGX-202 Earns Orphan Drug Status

Muscular Dystrophy News

RGX-202, a one-time experimental gene therapy for the treatment of Duchenne muscular dystrophy (DMD), has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA). The designation ... Read more