Neuromuscular Disease News and Research

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Patient-specific muscle models pave way for personalized neuromuscular therapies

Scientists have so far identified around 800 different neuromuscular diseases. These conditions are caused by problems in the way muscle cells, motor neurons and peripheral cells interact.

19 Dec 2023

First successful Phase 2 trial uses cutting-edge RNA CAR-T therapy to treat autoimmunity

Cartesian Therapeutics, a clinical-stage biotechnology company pioneering cell therapies for autoimmune diseases, announced today the publication of a landmark paper in The Lancet Neurology.

22 Jun 2023

Using human iPSC-derived cells to revolutionize the field of neurodegenerative disease

In this interview, we speak to Axol Bioscience about their human iPSC-derived cells and how they have the potential to revolutionize the field of neurodegenerative disease.

From Axol Bioscience Ltd 28 Nov 2022

Vaccines and monoclonal antibodies being developed for respiratory syncytial virus

A new study reviews recent advancements made in the development of RSV immunoprophylactic measures.

11 Aug 2022

UT Southwestern researchers reveal the physical mechanism behind a neurological autoimmune disease

Using UT Southwestern's Cryo-Electron Microscopy Facility, researchers for the first time have captured images of an autoantibody bound to a nerve cell surface receptor, revealing the physical mechanism behind a neurological autoimmune disease.

11 Jul 2022

New consensus-based recommendations designed to improve quality of life for DMD patients

Many more adults with Duchenne muscular dystrophy (DMD) are living longer thanks to improvements in treatment, however international standards of care have not yet fully addressed the complex needs of these patients.

9 Dec 2021

Identifying an important protein for motor coordination

News-Medical speaks to Professor Jorge Ruas about how he has identified an important protein involved in motor coordination.

10 Nov 2021

Study reveals the role of SPOP protein in an inherited form of ALS

The damaging effects of toxic proteins created in one inherited form of amyotrophic lateral sclerosis (ALS) are mediated by a protein called SPOP, according to a study published in the Proceedings of the National Academy of the Sciences.

8 Nov 2021

A new 3D model for myotonic dystrophy

Myotonic dystrophy is a hereditary degenerative neuromuscular disease that occurs mainly in adults, affecting about 50,000 people only in Spain.

4 May 2021

Spark Therapeutics launches gene therapy clinical trial for late-onset Pompe disease

Pompe disease is a hereditary genetic disorder caused by a deficiency of acid alpha-glucosidase leading to build-up of glycogen in the lyosomes, which then causes cell damage in various tissues, in particular the heart, the muscles, the liver and the nervous system.

3 Feb 2021

Canadian Neuromuscular Disease Registry increases patient access to research, clinical trials

The Canadian Neuromuscular Disease Registry (CNDR) was launched in 2010 to increase efficient patient access to cutting-edge research and clinical trials, to increase understanding of the natural history and epidemiology of neuromuscular disease across Canada, and to facilitate research collaboration.

3 Feb 2021

Genethon to start international clinical trial for treatment of Duchenne muscular dystrophy

Genethon, dedicated to designing and developing gene therapy products for rare diseases, received this Monday 30th of November the authorization from the ANSM, the French National Agency for Medicines and Health Products Safety, to start in France a multicentre international clinical trial for the treatment of Duchenne muscular dystrophy with product GNT 004.

2 Dec 2020

Researchers identify new strategy to combat loss of skeletal muscle mass

Researchers at the University of São Paulo (USP) in Brazil have shown for the first time that stimulating expression of a protein naturally produced by the human body can be a strategy to combat loss of skeletal muscle mass, which happens normally with aging but can intensify in cases of neurodegenerative or inflammatory disease, or in patients who spend long periods in intensive care units (ICUs).

9 Nov 2020

Study offers renewed hope for children suffering from myofibrillar myopathies

Stephen Greenspan and Laura Zah were devastated when they learned their son Alexander had a rare genetic mutation, which causes a deadly neuromuscular disease with no known treatment or cure.

14 Oct 2020

New drug offers hope for young boys with Duchenne muscular dystrophy

A new drug offers hope for young boys with the progressive neuromuscular disease Duchenne muscular dystrophy (DMD) by potentially offering an alternative to high-dose glucocorticoids that have significant side effects.

25 Sep 2020

Study implicates new gene as driver of motor neuron diseases

Failures in a quality control system that protects protein-building fidelity in cells can lead to motor neuron degeneration and related diseases, according to a new study from an international team co-directed by Scripps Research molecular biologist Claudio Joazeiro, PhD.

16 Sep 2020

Researchers find link between dystrophic muscles and lymphatic system in mice with Duchenne disease

Researchers at the Universities of Maynooth and Bonn discover a new connection in muscular dystrophy Duchenne muscular dystrophy (DMD) is the most common muscle disease in children and is passed on by X-linked recessive inheritance.

27 Aug 2020

Study examines safety and early outcomes of gene therapy for spinal muscular atrophy

In May 2019, the U.S. Food and Drug Administration approved a gene replacement therapy for the inherited, progressive neuromuscular disease 5q-linked spinal muscular atrophy.

25 Aug 2020

Study fills crucial knowledge gaps on myasthenia gravis

A study of 181 patients at 16 sites across the country who test negative for two antibodies long known to cause muscle-weakening myasthenia gravis, found that about 15% test positive for one of two newly discovered antibodies that also attack the point of communication between nerves and muscle.

19 Aug 2020

UOttawa's Faculty of Medicine awards 58 clinical research chairs to top clinician-scientists

The Faculty of Medicine has awarded 58 clinical research chairs to top clinician-scientists in a five-year, $40 million investment to drive clinical research excellence and enhance health and patient care.

30 Jul 2020