Muscular Dystrophy News

June 21, 2021June 21, 2021

EveryLife Introduces First of Kind ‘Roadmap’ to ICD Codes

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 ... Read more

June 18, 2021June 18, 2021

Get Ready for NORD’s Patient and Family Forum, Set for June 26-27

COVID-19 News, Muscular Dystrophy News

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 ... Read more

June 17, 2021June 18, 2021

It’s Year 15 for Jared Sweet Golf Outing DMD Fundraiser

Muscular Dystrophy News

The 15th Annual Jared Sweet Golf Outing, a Michigan event that benefits Parent Project Muscular Dystrophy (PPMD) and raises needed funds for research, is set for Saturday, June 19. To ... Read more

June 15, 2021June 15, 2021

Cholesterol Metabolism Is Potential Therapeutic Target in DMD

Muscular Dystrophy News

An analysis of microRNA molecules isolated from the blood of people with Duchenne muscular dystrophy (DMD) found alterations related to cholesterol metabolism, a study demonstrated. These findings suggest that the ... Read more

June 10, 2021June 10, 2021

DMD’s Course With Corticosteroid Use Examined in Review Study

Muscular Dystrophy News

With current corticosteroid use, people with Duchenne muscular dystrophy (DMD) typically lose their walking ability by their early teens, often need ventilation by their late teens, and usually survive until ... Read more

June 8, 2021June 8, 2021

Ionis Molecule Shows Early Promise for Myotonic Dystrophy Type 1

Muscular Dystrophy News

High levels of the toxic RNA molecule associated with myotonic dystrophy type 1 (DM1) lead to defects in muscle regeneration and in the number of muscle stem cells — or satellite ... Read more

June 7, 2021June 7, 2021

Rare 2030 Action Campaign Pushing for European Plan for Rare Diseases

Muscular Dystrophy News

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in ... Read more

June 4, 2021June 4, 2021

Advocates Can Try for Grants Ahead of Rare Disease Week on Capitol Hill

Muscular Dystrophy News

Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in ... Read more

June 3, 2021June 3, 2021

Vamorolone Safely Improves Muscle Function in DMD Boys, Trial Finds

Muscular Dystrophy News

Top-line data from the VISION-DMD Phase 2b clinical trial evaluating vamorolone in boys with Duchenne muscular dystrophy (DMD), showed efficacy across primary and secondary study goals. The investigational therapy was also ... Read more

June 1, 2021June 1, 2021

CRISPR Gene Editing Therapy Is Promising in New Mixed Model

Muscular Dystrophy News

A CRISPR-Cas9-based gene editing therapy promoted the production of a smaller but functional version of the dystrophin protein in a new mixed mouse model that uses muscle cells derived from ... Read more

May 27, 2021May 27, 2021

Gene Therapy Trial for Rare Form of MD Planned for Next Year

Muscular Dystrophy News

Asklepios BioPharmaceutical (AskBio) is planning to launch a Phase 1/2 clinical study to evaluate LION-101, its investigational gene therapy for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). Dosing in the trial ... Read more

May 25, 2021May 25, 2021

1st DMD Mouse Model to Mimic Heart Disease Confirms Gene Therapy Efficacy

Muscular Dystrophy News

For the first time, a new mouse model has been shown to mimic the heart disease features — specifically, to progress into late-stage heart failure, similar to humans — of ... Read more

May 20, 2021May 20, 2021

DMD Gene Therapy, SRP-9001, Showing ‘Robust’ Micro-dystrophin in Trial

Muscular Dystrophy News

Sarepta Therapeutics‘ investigational gene therapy SRP-9001 showed early signs of biologic efficacy in muscle biopsies from 11 boys with Duchenne muscular dystrophy (DMD) treated in a Phase 1 clinical trial, the ... Read more

May 18, 2021May 18, 2021

Two More Patients Dosed in Solid Trial of DMD Gene Therapy SGT-001

Muscular Dystrophy News

Two more participants have been dosed in the IGNITE DMD clinical trial, bringing to eight the total number of Duchenne muscular dystrophy (DMD) patients thus far given Solid Biosciences‘ investigational ... Read more

May 13, 2021May 13, 2021

FDA Puts Investigational FSHD Therapy Losmapimod on Fast Track

Muscular Dystrophy News

The U.S. Food and Drug Administration (FDA) has granted fast track designation to losmapimod, a potential treatment for facioscapulohumeral muscular dystrophy (FSHD) being developed by Fulcrum Therapeutics. The designation is ... Read more