Muscular Dystrophy News and Research

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The muscular dystrophies (MD) are a group of more than 30 genetic diseases characterized by progressive weakness and degeneration of the skeletal muscles that control movement. Some forms of MD are seen in infancy or childhood, while others may not appear until middle age or later. The disorders differ in terms of the distribution and extent of muscle weakness (some forms of MD also affect cardiac muscle), age of onset, rate of progression, and pattern of inheritance.

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

UAB study identifies new molecule for treating hypertension

Researchers at the UAB have carried out two revealing studies on TRPV2, a key ion channel in several cellular functions, which points to it as a possible new therapeutic target in the treatment of hypertension.

6 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

Calcium channel blockers reverse muscle weakness in animal model of myotonic dystrophy type 1

New research has identified the specific biological mechanism behind the muscle dysfunction found in myotonic dystrophy type 1 (DM1) and further shows that calcium channel blockers can reverse these symptoms in animal models of the disease.

2 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

Tiny amounts of hydrogen sulfide could help people live healthier for longer

Future therapies to help people live healthy lives for longer could be developed from drugs that release tiny amounts of the gas hydrogen sulfide (H2S), new research has indicated.

31 Jul 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

AI may hold the key to a sharper and more precise DNA scissor technology

Artificial Intelligence may be the key to more precise and effective gene therapy treatments. A new study from Aarhus University has found, that applying AI predictions of protein structures enhances the DNA scissor technology CRISPR, by making the cuts in a patient's DNA more precise.

29 Jun 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

New approach improves diagnosis of limb-girdle muscular dystrophy

It's not easy to distinguish between the dozens of subtypes of limb girdle muscular dystrophy -; a rare, genetic muscle disease characterized by weakness in the hips and shoulders that causes difficulty walking and lifting the arms.

15 Jun 2023