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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.
New study opens door to preventative treatments for congenital diaphragmatic hernia

New study opens door to preventative treatments for congenital diaphragmatic hernia

Congenital diaphragmatic hernia (CDH) is not as well known as muscular dystrophy and cystic fibrosis, but like them it is a life-threatening birth defect, and is just as common. Occurring in one in 3,000 births, CDH causes the guts and liver to protrude through a defective diaphragm and into the chest cavity, where they interfere with the lungs. [More]
UVA researcher identifies potential therapeutic target for myotonic muscular dystrophy

UVA researcher identifies potential therapeutic target for myotonic muscular dystrophy

A doctor who was one of the discoverers of the gene responsible for myotonic muscular dystrophy has now identified a therapeutic that could modify progression of muscle damage and muscle dysfunction associated with the disease - issues that cause patients significant disability and deterioration in quality of life. [More]
Quest Diagnostics to provide whole exome sequencing service to diagnose neurological disorders

Quest Diagnostics to provide whole exome sequencing service to diagnose neurological disorders

Quest Diagnostics, the world's leading provider of diagnostic information services, today announced the availability of Neurome, a whole exome sequencing service designed to aid the diagnosis of rare neurological disorders in pediatric populations. [More]
TWi Biotechnology obtains Rare Disease Drug designation in Taiwan for use of AC-203 to treat EBS

TWi Biotechnology obtains Rare Disease Drug designation in Taiwan for use of AC-203 to treat EBS

TWi Pharmaceuticals, Inc. today announced that its fully owned subsidiary, TWi Biotechnology, Inc., has received the designation of Rare Disease Drug by Taiwan FDA for use of AC-203 to treat Epidermolysis Bullosa Simplex (EBS), and is eligible for applying for coverage under National Health Insurance Administration. [More]
Researchers reveal role of abdominal muscles in muscular dystrophy process

Researchers reveal role of abdominal muscles in muscular dystrophy process

The muscular dystrophies are known to target various muscle groups differentially. In addition to making limb muscles weak, muscular dystrophy (MD) can also lead to decreased function of specific muscles involved in respiration causing breathing difficulties as well as leading to cardiac problems. [More]
Researchers generate mature, functional skeletal muscles using new approach

Researchers generate mature, functional skeletal muscles using new approach

A team of researchers from Italy, Israel and the United Kingdom has succeeded in generating mature, functional skeletal muscles in mice using a new approach for tissue engineering. The scientists grew a leg muscle starting from engineered cells cultured in a dish to produce a graft. [More]
Duke researchers demonstrate genetic therapeutic technique to treat DMD patients

Duke researchers demonstrate genetic therapeutic technique to treat DMD patients

Duke researchers have demonstrated a genetic therapeutic technique that has the potential to treat more than half of the patients suffering from Duchenne Muscular Dystrophy (DMD). [More]
EC grants orphan drug designation to aTyr Pharma's Resolaris for treatment of FSHD

EC grants orphan drug designation to aTyr Pharma's Resolaris for treatment of FSHD

aTyr Pharma, Inc., a biotherapeutics company engaged in the discovery and development of Physiocrine-based therapeutics to address rare diseases, announced today the European Commission (EC) has granted orphan drug designation to Resolaris for the treatment of facioscapulohumeral muscular dystrophy (FSHD). [More]
Public health researchers report frequency of two muscle-weakness disorders

Public health researchers report frequency of two muscle-weakness disorders

Researchers in public health have reported in the first broad study in the United States the frequency of two muscle-weakness disorders that strike mostly boys: Duchenne muscular dystrophy and Becker muscular dystrophy. [More]
First human clinical study of ChromaDex's NIAGEN nicotinamide riboside meets primary endpoint

First human clinical study of ChromaDex's NIAGEN nicotinamide riboside meets primary endpoint

ChromaDex Corp. announced today that the initial results of the first human clinical study for the company's NIAGEN nicotinamide riboside (NR) has met its primary endpoint. [More]
UI Children's Hospital named Certified Duchenne Care Center by PPMD

UI Children's Hospital named Certified Duchenne Care Center by PPMD

The University of Iowa Children's Hospital was named a Certified Duchenne Care Center by Parent Project Muscular Dystrophy, a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne) and demanding optimal care for all people with Duchenne. [More]
Study demonstrates therapeutic potential of new class of synthetic oligonucleotides for DMD treatment

Study demonstrates therapeutic potential of new class of synthetic oligonucleotides for DMD treatment

Research efforts associating scientists from the CNRS, UVSQ and INSERM within the Laboratoire END-ICAP, working in collaboration with a team from the University of Bern, has demonstrated the therapeutic potential of a new class of synthetic oligonucleotides in the treatment of Duchenne muscular dystrophy (DMD) using RNA "surgery". [More]
Bern researchers develop active substance for treating Duchenne muscular dystrophy

Bern researchers develop active substance for treating Duchenne muscular dystrophy

Duchenne muscular dystrophy is a congenital disease which causes muscle degeneration and eventual death in teenagers. Recently, researchers from Bern developed an active substance, which they together with an international team tested successfully. [More]
MRIGlobal awarded $54 million NIH contract to provide support for drug development

MRIGlobal awarded $54 million NIH contract to provide support for drug development

MRIGlobal today announced that it has been awarded a $54 million, 10-year program from the National Institute of Neurological Disorders and Stroke to provide support for the development and manufacturing of drug candidates for ultimate use in human clinical trials. [More]
Duke researchers grow contracting human skeletal muscle in lab

Duke researchers grow contracting human skeletal muscle in lab

In a laboratory first, Duke researchers have grown human skeletal muscle that contracts and responds just like native tissue to external stimuli such as electrical pulses, biochemical signals and pharmaceuticals. [More]
New way to measure upper extremity movement in muscular dystrophy patients

New way to measure upper extremity movement in muscular dystrophy patients

Researchers at Nationwide Children's Hospital have developed a way to measure upper extremity movement in patients with muscular dystrophy using interactive video game technology. Their hope is to expand inclusion criteria for clinical trials to incorporate patients using wheelchairs. [More]
Neurological testing accessibility and affordability: an interview with Dr Joseph Higgins

Neurological testing accessibility and affordability: an interview with Dr Joseph Higgins

Neurological disorders with genetic causes can be very difficult to diagnose without reliable, clinically relevant tests tailored to specific phenotypes. [More]
Early use of heart failure drugs slows cardiac damage in boys, young men with DMD

Early use of heart failure drugs slows cardiac damage in boys, young men with DMD

Early use of available heart failure drugs slows the progressive decline in heart function before symptoms are apparent in boys and young men with Duchenne muscular dystrophy (DMD), according to a new study published online by The Lancet Neurology. [More]
PTC begins rolling NDA submission for Translarna to treat nmDMD

PTC begins rolling NDA submission for Translarna to treat nmDMD

PTC Therapeutics, Inc. today announced that it has commenced a rolling submission of a New Drug Application to the United States Food and Drug Administration for Translarna™ for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). [More]
Isis Pharmaceuticals begins ISIS-DMPK Rx clinical study in DM1 patients

Isis Pharmaceuticals begins ISIS-DMPK Rx clinical study in DM1 patients

Isis Pharmaceuticals, Inc. announced today that it has initiated a study for ISIS-DMPK Rx in patients with Myotonic Dystrophy Type 1 (DM1). DM1 is a rare genetic neuromuscular disease caused by the production of toxic dystrophia myotonica-protein kinase (DMPK) RNA in cells. ISIS-DMPKRx is specifically designed to reduce toxic DMPK RNA. [More]
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