Neuromuscular Disease News and Research

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Home-based pulmonary function monitoring for teens with Duchenne muscular dystrophy

Heart and lung complications are responsible for much of the morbidity and mortality associated with Duchenne muscular dystrophy (DMD).

18 Dec 2018

Genetic testing and breakthrough therapies may transform diagnosis, care of neuromuscular disease

A new report funded by the Muscular Dystrophy Association and released by the IQVIA Institute for Human Data Science indicates that genetic testing and breakthrough therapies will transform the diagnosis and care of neuromuscular disease within the next decade.

31 Oct 2018

Genentech announces clinical data from studies investigating risdiplam in spinal muscular atrophy

Genentech, a member of the Roche Group, announced today interim clinical data from the dose-finding parts of the pivotal FIREFISH and SUNFISH studies investigating risdiplam in spinal muscular atrophy.

3 Oct 2018

Researchers discover how nerve cells control movement

Researchers at Karolinska Institutet in Sweden have discovered a new way in which nerve cells can control movement. In a study on zebrafish published in the journal PNAS they show that the contact between neurons and muscles is more dynamic than previously thought.

3 Oct 2018

Alternative method identified for delivering nusinersen to spinal muscular atrophy patients

A new report has identified an alternative method to deliver nusinersen to patients with spinal muscular atrophy using a subcutaneous intrathecal catheter system configured by connecting an intrathecal catheter to an implantable infusion port.

24 Aug 2018

MDA announces 34 new grants totaling $9.9 million to accelerate neuromuscular research

The Muscular Dystrophy Association today announced the award of 34 new grants totaling more than $9.9 million for its summer round of funding. These new grants represent a continued commitment by MDA to fund groundbreaking research that will accelerate treatments and cures for the more than 40 diseases in its program.

24 Aug 2018

Researchers discover new approach for treatment of myotonic dystrophy

Researchers at the Translational Genomics laboratory of Valencia University and the INCLIVA health research institute have just discovered a new approach for the treatment of myotonic dystrophy, a rare and incurable neuromuscular disease which does not have specific treatment.

27 Jun 2018

KFU medical departments seek to find treatments for hereditary syndromes

Newest results were showcased at the International Myology School in Moscow on 16th - 19th May 2018. KFU was represented by Junior Research Associate Mikhail Mavlikeev.

6 Jun 2018

Beaumont and GVSU team up to develop lifesaving cough-assist device

The act of coughing may seem annoying, but for those who can no longer cough on their own because of a medical condition, it's a matter of life and death. Today, more people are surviving brain/spinal cord injuries caused by stroke and automobile accidents.

7 May 2018

Study identifies potential genetic link in sudden infant death syndrome

Rare genetic mutations associated with impairment of the breathing muscles are more common in children who have died from sudden infant death syndrome (also known as ‘cot death’) than in healthy controls, suggesting a possible genetic element of the disorder, according to a case-control study in the UK and USA published in The Lancet.

29 Mar 2018

Cell therapy for heart disease caused by muscular dystrophy also improves limb strength, study shows

Injections of cardiac progenitor cells help reverse the fatal heart disease caused by Duchenne muscular dystrophy and also lead to improved limb strength and movement ability, a new study shows.

23 Feb 2018

Researchers describe human blood-nerve barrier transcriptome for the first time

Human peripheral nerves -- all the nerves outside of the central nervous system -- are protected by the blood-nerve barrier. This is a tight covering of endothelial cells that maintains the microenvironment within the nerves by restricting the amounts or types of water, ions, solutes and nutrients that can reach the axons, or electric cables within the nerves, from the blood circulation system.

8 Feb 2018

New, sensitive screening test may allow clinicians to accurately detect Pompe disease

Researchers identified a new, more sensitive screening test to recognize Pompe disease, a metabolic disorder affecting cellular processing of glycogen in numerous tissues of the body.

2 Jan 2018

Clinical trial results could lead to treatment of heart disease in Duchenne muscular dystrophy patients

After boys and young men with Duchenne muscular dystrophy received cardiac progenitor cell infusions, medical tests indicated that the patients' hearts appeared improved, results from a new study show. Patients in the study also scored higher on arm strength tests after receiving the cell infusions.

15 Nov 2017

MDA announces 13 new research grants to advance treatment for neuromuscular diseases

The Muscular Dystrophy Association today announced 13 new research and development grants totaling $3.5 million to accelerate the development of treatments for muscular dystrophy, ALS and related life-threatening diseases.

14 Nov 2017

Preclinical study demonstrates efficacy of gene therapy in treatment of Duchenne muscular dystrophy

Researchers from Genethon, the AFM-Telethon laboratory, Inserm (UMR 1089, Nantes) and the University of London (Royal Holloway) demonstrated the efficacy of an innovative gene therapy in the treatment of Duchenne muscular dystrophy.

25 Jul 2017

Millions of kids fall outside Senate plan to shield disabled from Medicaid cuts

Aidan Long is a 13-year-old from Kalispell, Mont., who has suffered multiple daily seizures since he was 4. The seizures defy medical cure, and some of them continue for weeks, requiring Aidan to be airlifted to children's hospitals in Denver or Seattle, said his father, Ben Long.

10 Jul 2017

Neuromuscular Disease News and Research

Home-based pulmonary function monitoring for teens with Duchenne muscular dystrophy

Genetic testing and breakthrough therapies may transform diagnosis, care of neuromuscular disease

Genentech announces clinical data from studies investigating risdiplam in spinal muscular atrophy

Researchers discover how nerve cells control movement

Alternative method identified for delivering nusinersen to spinal muscular atrophy patients

MDA announces 34 new grants totaling $9.9 million to accelerate neuromuscular research

Researchers discover new approach for treatment of myotonic dystrophy

KFU medical departments seek to find treatments for hereditary syndromes

Beaumont and GVSU team up to develop lifesaving cough-assist device

Study identifies potential genetic link in sudden infant death syndrome

Cell therapy for heart disease caused by muscular dystrophy also improves limb strength, study shows

Researchers describe human blood-nerve barrier transcriptome for the first time

New, sensitive screening test may allow clinicians to accurately detect Pompe disease

Clinical trial results could lead to treatment of heart disease in Duchenne muscular dystrophy patients

MDA announces 13 new research grants to advance treatment for neuromuscular diseases

Preclinical study demonstrates efficacy of gene therapy in treatment of Duchenne muscular dystrophy

Millions of kids fall outside Senate plan to shield disabled from Medicaid cuts

Honda gives $1 million gift to Nationwide Children’s Hospital to enhance pediatric mobility

Targeting RNA repeats

Gene therapy treats myotubular myopathy in dogs

Neuroscience - Second Edition eBook

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

From Puberty to Menopause: Clue’s CEO, Audrey Tsang on the Power of Femtech

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