Duchenne Muscular Dystrophy News and Research

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Duchenne muscular dystrophy (DMD) is a progressive muscle disorder that causes the loss of both muscle function and independence. DMD is perhaps the most prevalent of the muscular dystrophies and is the most common lethal genetic disorder diagnosed during childhood today. Each year, approximately 20,000 children worldwide are born with DMD (one of every 3,500 male children).

Adeno-associated virus: The gene therapy revolution faces manufacturing and safety hurdles

Recombinant adeno-associated virus (rAAV)-based genetic applications to treat human diseases.

5 Apr 2024

Lurie Children's Hospital administers first gene therapy for Duchenne muscular dystrophy in Illinois

On March 27, 2024, Ann & Robert H. Lurie Children's Hospital of Chicago treated its first patient with ELEVIDYS (delandistrogene moxeparvovec-rokl), the first gene therapy for Duchenne muscular dystrophy – a rare, genetic disease characterized by progressive muscle damage and weakness.

3 Apr 2024

Breakthrough in CRISPR delivery promises safer gene editing

Recent research in PNAS highlights advancements in non-viral, cell-specific delivery methods for CRISPR-Cas systems, emphasizing their potential to enhance research and gene therapy applications by minimizing risks associated with off-target effects and improving targeted delivery.

7 Mar 2024

Key gene found to prevent muscle breakdown in muscular dystrophy

A specific gene may play a key role in new treatments that prevent muscle in the body from breaking down in serious muscle diseases, muscular dystrophies.

6 Mar 2024

Unbiased assessment of vamorolone for treating Duchenne muscular dystrophy

The drug vamorolone (Agamree) has been hailed as a promising new drug to treat Duchenne muscular dystrophy (DMD).

25 Jan 2024

UCI researchers discover gene critical for muscle repair

University of California, Irvine researchers have identified a gene expressed during regeneration that is critical for muscle repair.

12 Nov 2023

C-Path releases new publication on transforming drug development for neurological disorders

Critical Path Institute (C-Path) is pleased to announce the release of a new peer-reviewed publication, titled "Transforming Drug Development for Neurological Disorders: Proceedings from a Multi-disease Area Workshop," now published in Neurotherapeutics, The Journal of the American Society for Experimental Neurotherapeutics.

9 Nov 2023

New drug gets FDA approval for the treatment of common genetic disease in young boys

A new drug developed by professors from the School of Pharmacy and Pharmaceutical Sciences at Binghamton University has received Food and Drug Administration (FDA) approval for the treatment of patients with Duchenne muscular dystrophy (DMD), a common genetic disease that mostly affects young boys.

31 Oct 2023

FDA approves a new steroidal-type anti-inflammatory drug for Duchenne muscular dystrophy

Boys with Duchenne muscular dystrophy (DMD) have a clinically proven, new treatment option with the Food and Drug Administration's approval Thursday of vamorolone, a steroidal-type, anti-inflammatory drug developed based on research performed at Children's National Hospital.

27 Oct 2023

Dual CRISPR-Cas3 is a promising tool to induce a gigantic genomic deletion and restore dystrophin protein

Researchers evaluated the use of a dual clustered regularly interspaced short palindromic repeats (CRISPR)-Cas3 system.

29 Aug 2023

Next-generation sequencing provides novel insights into the mechanisms underlying autism and myotonic muscular dystrophy

The pathomechanistic connection between autism and myotonic muscular dystrophy type 1.

16 Aug 2023

Duchenne and other dystrophinopathies research receives major boost from new collaboration

Critical Path Institute's (C-Path) Duchenne Regulatory Science Consortium (D-RSC) and the Duchenne Data Foundation (DDF) are excited to announce a joint collaboration aimed at advancing research and improving healthcare for individuals with Duchenne muscular dystrophy (DMD) and other dystrophinopathies (conditions linked to mutations in the DMD gene).

10 Aug 2023

Nationwide Children's Hospital pioneers new gene therapy for Duchenne muscular dystrophy

In a landmark moment for the Abigail Wexner Research Institute at Nationwide Children's, a 5-year-old from Bellefontaine, Ohio, received the first dose of a recently approved gene therapy for Duchenne muscular dystrophy at Nationwide Children's Hospital, where the therapy was invented and initially tested.

8 Aug 2023

New porcine model could help develop treatments for Dduchenne muscular dystrophy

The group of LMU scientist Eckhard Wolf has developed a porcine DMD model with a mutation, which mimics the hallmarks of the human disease but develops them in an accelerated mode.

12 Jul 2023

Alternative splicing in neurodegenerative disease and the promise of RNA-based therapeutic strategies

Alternative splicing, a clever way a cell generates many different variations of messenger RNAs -; single-stranded RNAs involved in protein synthesis -; and proteins from the same stretch of DNA, plays an important role in molecular biology.

21 Jun 2023

Manipulating a signaling protein to slow down muscular dystrophy

A team of researchers at the University of Houston College of Pharmacy is reporting that by manipulating TAK1, a signaling protein that plays an important role in development of the immune system, they can slow down disease progression and improve muscle function in Duchenne muscular dystrophy (DMD).

25 May 2023

Scarring to the collagen framework causes dysfunction in Duchenne muscular dystrophy

Muscles that ache after a hard workout usually don't hurt for long, thanks to stem cells that rush to the injured site along "collagen highways" within the muscle and repair the damaged tissue.

11 May 2023

Novel molecule may help overcome the devastating symptoms of Duchenne muscular dystrophy

In a new study published in The FASEB Journal, investigators demonstrated the potential of a molecule that may help overcome some of the devastating symptoms of Duchenne muscular dystrophy (DMD), the most common life-limiting congenital neuromuscular disorder.

5 Apr 2023

Rare Diseases Action Plan for England focuses on providing better care and reducing health inequalities

A new Rare Diseases Action Plan for England will be published today (Tuesday 28 February) that will ensure those living with these conditions receive better care and treatment, fairer access to testing and have continued support.

28 Feb 2023

Oxford-Harrington Rare Disease Centre hosts virtual event in honor of the upcoming Rare Disease Day

The Oxford-Harrington Rare Disease Centre, a partnership of the University of Oxford and Harrington Discovery Institute at University Hospitals, is hosting a virtual event on Monday, Feb. 27, 2023, in honor of the upcoming Rare Disease Day, which is celebrated across the globe on February 28 every year.

21 Feb 2023