FDA Fast Tracks Regenxbio Gene Therapy
The FDA granted Regenxbio RGX-202, a prospective gene therapy for Duchenne Muscular Dystrophy, a Fast Track designation.
Category: Muscular Dystrophy
Muscular Dystrophy
Latest
Muscular Dystrophy
Latest
Call for Projects to Find Treatments for Duchenne Muscular Dystrophy
Parent Project Muscular Dystrophy and Duchenne UK launch their 2022 Joint Call for Therapeutic Projects inviting research proposals.
Muscular Dystrophy
Latest
Blocking an Ion Channel Improves Muscle Function and Survival in Mice with Severe Duchenne Muscular Dystrophy
A drug first developed to treat kidney disease prolongs survival and improves muscle function in mice with Duchenne, Johns Hopkins reports.
Oxford-Harrington Rare Disease Centre to Advance Novel Treatment for Duchenne Muscular Dystrophy
The Oxford-Harrington Rare Disease Centre announce its support of a novel treatment for Duchenne muscular dystrophy.
Read More
Sarepta Therapeutics Intends to Submit an Accelerated BLA for Its Gene Therapy to Treat Duchenne Muscular Dystrophy
Sarepta Therapeutics Inc announces its intent to submit a BLA seeking accelerated approval for SRP-9001 to treat individuals with Duchenne.
Read More
Bionic Clothing Maker CIONIC Collaborates with Yves Behar’s fuseproject
CIONIC announces a collaboration with acclaimed designer Yves Behar and his world-renowned multidisciplinary design firm, fuseproject.
Read More
Parent Project Muscular Dystrophy Certifies Pittsburgh Clinic
Parent Project Muscular Dystrophy adds the clinic at UPMC Children’s Hospital of Pittsburgh to its Certified Duchenne Care Center Program.
Read More
Fulcrum Phase 3 REACH FSHD Clinical Trial Using AMRA Medical’s MRI Measurements
Fulcrum Therapeutics is using AMRA Medical’s muscle fat infiltration (MFI) as a secondary endpoint in its phase 3 FSHD clinical trial: REACH.
Read More
Parent Project Muscular Dystrophy (PPMD) Announces Pediatric Certified Duchenne Care Center at St Louis Children’s Hospital
Parent Project Muscular Dystrophy expands its Certified Duchenne Care Program with the certification of the first clinic in St. Louis.
Read More
How Does Duchenne Muscular Dystrophy Cause Heart Rhythm Problems?
Mutations that cause Duchenne muscular dystrophy slow electrical activity in the heart, causing abnormal heart rhythms, a study notes.
Read More
AMRA Medical Continues to Advance FSHD Research with Whole-Body Measurements
AMRA Medical publishes results of its Fulcrum-sponsored study highlighting the development and use of a MRI protocol for muscle analysis.
Read More
Parent Project Muscular Dystrophy Nominates Duchenne for RUSP
Parent Project Muscular Dystrophy nominates Duchenne to be part of Recommended Uniform Screening Panel (RUSP) for newborns.
Read More
LGMD Community Celebrates Adoption of ICD-10 Diagnostic Codes for LGMD
ICD-10-CM codes for LGMD and several of its subtypes will be fully implemented and ready for use starting this October, MDA reports.
Read More
Cancer Drug Shows Potential as Treatment for Muscular Dystrophy
An existing cancer drug could have potential as a treatment for muscular dystrophy, researchers suggest in a new study.
Read More
Parent Project Muscular Dystrophy Hosts 28th Annual International Conference in Scottsdale, Ariz
Parent Project Muscular Dystrophy will host its 2022 Annual Conference in Scottsdale, Arizona, June 23-26.
Read More
Avidity Biosciences Supports World FSHD Day and Presents Preclinical Data from Program
Avidity Biosciences Inc joins in activities to raise awareness for FSHD and highlights preclinical treatment results supporting AOC 1020.
Read More
FSHD Society Convenes in Florida in June
The FSHD Society is holding back-to-back meetings in June in Orlando to help develop treatments for facioscapulohumeral muscular dystrophy.
Read More
Gamers: MDA Rivals Streaming Event Takes Place June 18
MDA Rivals on Saturday, June 18, aims to support funding for people living with muscular dystrophy, ALS, and related neuromuscular diseases.
Read More