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.

Researchers find method to repair gene mutation causing autoimmune deficiency disease

Researchers at Karolinska Institutet in Sweden have found a method to repair the gene mutation causing agammaglobulinemia, an autoimmune deficiency disease that almost exclusively affects boys and in which the body lacks the ability to produce immunoglobulins (gamma globulin).

14 Aug 2014

Novogen receives funding support to commence studies in muscular dystrophy

The Australian biotechnology company, Novogen Limited today announced receipt of funding from the FSHD Global Research Foundation as part of the Company’s efforts to find effective treatments for a range of musculo-degenerative diseases including facioscapulohumeral dystrophy (FSHD).

11 Aug 2014

Scientists identify mechanism by which inherited neurological disease causes muscle weakness in men

Researchers at University of California, San Diego School of Medicine have identified the mechanism by which a rare, inherited neurodegenerative disease causes often crippling muscle weakness in men, in addition to reduced fertility.

11 Aug 2014

Isoform: New protein offers novel therapeutic approach for patients with DMD

Scientists have discovered a new form of dystrophin, a protein critical to normal muscle function, and identified the genetic mechanism responsible for its production.

11 Aug 2014

Small molecule may be able to convince damaged nerves to effectively rewire circuits

Frogs, dogs, whales, snails can all do it, but humans and primates can't. Regrow nerves after an injury, that is—while many animals have this ability, humans don't.

6 Aug 2014

UTHealth scientists identify inhibitory switch to prevent peripheral vascular disease

Millions of people in the United States have a circulatory problem of the legs called peripheral vascular disease. It can be painful and may even require surgery in serious cases. This disease can lead to severe skeletal muscle wasting and, in turn, limb amputation.

25 Jul 2014

Splice-switching oligonucleotide drugs alter editing of gene transcript

In splice-switching, an innovative therapeutic approach, targeted oligonucleotide drugs alter the editing of a gene transcript to produce the desired form of a protein.

18 Jul 2014

PTC Therapeutics initiates reimbursed expanded access program for Translarna

PTC Therapeutics, Inc. today announced the initiation of a reimbursed expanded access program (EAP). PTC's EAP program is intended to make Translarna (ataluren) available to patients before commercial availability in certain countries.

9 Jul 2014

FDA grants Fast Track designation to Akashi Therapeutics' HT-100 for treatment of DMD

Akashi Therapeutics, Inc., announced today that the U.S. Food and Drug Administration has granted Fast Track designation to the company's most advanced product candidate, HT-100 (delayed-release halofuginone), an orally available, small molecule drug candidate intended to reduce fibrosis and inflammation and promote healthy muscle regeneration in boys with DMD.

3 Jul 2014

Isis Pharmaceuticals begins ISIS-DMPK Rx Phase 1 study for treatment of Myotonic Dystrophy Type 1

Isis Pharmaceuticals, Inc. announced today that it has initiated a Phase 1 study for ISIS-DMPKRx. Isis earned a $14 million milestone payment from Biogen Idec associated with this achievement.

9 Jun 2014

New gene involved in Parkinson's disease, find researchers

A team of UCLA researchers has identified a new gene involved in Parkinson's disease, a finding that may one day provide a target for a new drug to prevent and potentially even cure the debilitating neurological disorder.

5 Jun 2014

Nationwide Insurance Foundation donates $10M to Nationwide Children's Hospital

Nationwide Children's Hospital today announced that it has received a $10 million gift from the Nationwide Insurance Foundation.

27 May 2014

EMA's CHMP gives positive opinion for PTC Therapeutics' Translarna

PTC Therapeutics, Inc. today announced that following its request for re-examination, the Committee for Medicinal Products for Human Use of the European Medicines Agency has adopted a positive opinion regarding the company's application for a conditional marketing authorization of Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged five years and older.

23 May 2014

DNA-directed RNA interference and hepatitis C: an interview with Carl Stubbings, Chief Business Officer, Benitec Biopharma

RNA interference (RNAi) is a naturally occurring process, which cells can use to silence, or ‘turn off’ unwanted genes.

From Quest Diagnostics 19 May 2014

Researchers identify heart-specific form of protein that protects against irregular heartbeats

Researchers at the Cedars-Sinai Heart Institute have identified a heart-specific form of a protein, BIN1, responsible for sculpting tiny folds in pockets that are present on the surface of heart muscle cells.

19 May 2014

Eli and Edythe Broad Foundation gift $4 million to support UCLA research

Two new gifts from The Eli and Edythe Broad Foundation to UCLA totaling $4 million will fund research in stem cell science and digestive diseases and support the recruitment of key faculty at two renowned research centers.

9 May 2014

Sildenafil may help boys suffering from muscular dystrophy disease

Drugs used to treat erectile dysfunction may be able to slow disease progression in boys who suffer from the muscle wasting disease Duchenne muscular dystrophy.

8 May 2014

Commonly used drug restores blood flow to oxygen-starved muscles of boys with muscular dystrophy

Cedars-Sinai Heart Institute researchers have found that a commonly prescribed drug restores blood flow to oxygen-starved muscles of boys with Duchenne muscular dystrophy, a genetic muscle-wasting disease that rarely is seen in girls but affects one in 3,500 male babies, profoundly shortening life expectancy. It is the most common fatal disease that affects children.

8 May 2014

Common drug may improve blood flow in muscles of boys with Duchenne muscular dystrophy

A drug typically prescribed for erectile dysfunction or increased pressure in the arteries may help improve blood flow in the muscles of boys with Duchenne muscular dystrophy, according to a study published in the May 7, 2014, online issue of Neurology®, the medical journal of the American Academy of Neurology.

8 May 2014

Novogen partners with Genea Biocells to accelerate testing for neurodegenerative diseases

Novogen Limited, an oncology drug development company, today announced a collaboration with Australian company, Genea Biocells, to accelerate testing of their super-benzopyran (SBP) drugs for degenerative diseases of the nervous system and muscles.

5 May 2014

Muscular Dystrophy News and Research

Further Reading

Researchers find method to repair gene mutation causing autoimmune deficiency disease

Novogen receives funding support to commence studies in muscular dystrophy

Scientists identify mechanism by which inherited neurological disease causes muscle weakness in men

Isoform: New protein offers novel therapeutic approach for patients with DMD

Small molecule may be able to convince damaged nerves to effectively rewire circuits

UTHealth scientists identify inhibitory switch to prevent peripheral vascular disease

Splice-switching oligonucleotide drugs alter editing of gene transcript

PTC Therapeutics initiates reimbursed expanded access program for Translarna

FDA grants Fast Track designation to Akashi Therapeutics' HT-100 for treatment of DMD

Isis Pharmaceuticals begins ISIS-DMPK Rx Phase 1 study for treatment of Myotonic Dystrophy Type 1

New gene involved in Parkinson's disease, find researchers

Nationwide Insurance Foundation donates $10M to Nationwide Children's Hospital

EMA's CHMP gives positive opinion for PTC Therapeutics' Translarna

DNA-directed RNA interference and hepatitis C: an interview with Carl Stubbings, Chief Business Officer, Benitec Biopharma

Researchers identify heart-specific form of protein that protects against irregular heartbeats

Eli and Edythe Broad Foundation gift $4 million to support UCLA research

Sildenafil may help boys suffering from muscular dystrophy disease

Commonly used drug restores blood flow to oxygen-starved muscles of boys with muscular dystrophy

Common drug may improve blood flow in muscles of boys with Duchenne muscular dystrophy

Novogen partners with Genea Biocells to accelerate testing for neurodegenerative diseases

Revolutionizing Life Science: An Interview with SCIEX on ASMS, the SCIEX 7500+ System, and AI-Driven Quantitation

From Discovery Biology to ELRIG Chair

Breathing New Life into Diagnostics: Plasmion's SICRIT Technology

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