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,…
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 date, the outing has raised more than $160,000 for the PPMD. Since the event’s inception, the Sweet family,…
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 cholesterol metabolic pathway may be a potential therapeutic target for this most common type of muscular dystrophy, the…
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 the third or fourth decade of life, according to a review study into the clinical course of DMD today.
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 cells — and promotes muscle fibrosis (scarring) upon injury, according to a study in a mouse model of DM1.
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 Europe living with rare diseases are left behind by the start of a new decade. As part of…
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 the week’s pre-events. The top 50 point-earners will be eligible to win $1,000 to $5,000 in grants, totaling…
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 well-tolerated with a…
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 Duchenne muscular dystrophy (DMD) patients. The therapy is designed to delete a DMD gene region that is commonly mutated in people…
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 is expected to start in the first half of next year. “We are excited to begin clinical trials…
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