Dystrophin News and Research RSS Feed - Dystrophin News and Research

Dystrophin is a rod-shaped cytoplasmic protein, and a vital part of a protein complex that connects the cytoskeleton of a muscle fiber to the surrounding extracellular matrix through the cell membrane.
New iPS cell model provides clear drug target for treatment of DMD

New iPS cell model provides clear drug target for treatment of DMD

Duchenne muscular dystrophy (DMD) is a muscular disease that shows symptoms in early childhood and causes progressive atrophy and eventual death. There is little in terms of treatment, partly because of poor understanding of how DMD develops, although it is known that abnormal expression of the protein dystrophin is at fault. [More]
Kay E. Davies named recipient of ASHG's 2015 William Allan Award

Kay E. Davies named recipient of ASHG's 2015 William Allan Award

The American Society of Human Genetics has named Kay E. Davies, DPhil, Dr. Lee's professor of anatomy, associate head of the medical sciences division; and director of the Medical Research Council Functional Genomics Unit in the department of physiology, anatomy and genetics at the University of Oxford, the 2015 recipient of the annual William Allan Award. [More]
University of Washington receives PPMD grant to continue analysis of spectrin-like repeats in dystrophin

University of Washington receives PPMD grant to continue analysis of spectrin-like repeats in dystrophin

Parent Project Muscular Dystrophy, a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne) awarded University of Washington a $148,000 grant to continue the functional analysis of spectrin-like repeats in dystrophin. [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]
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]
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]
Researchers show that iPS cells can be used to edit genetic mutations that cause DMD

Researchers show that iPS cells can be used to edit genetic mutations that cause DMD

Researchers at the Center for iPS Cell Research and Application, Kyoto University, show that induced pluripotent stem (iPS) cells can be used to correct genetic mutations that cause Duchenne muscular dystrophy (DMD). [More]
PPMD, Santhera Pharmaceuticals collaborate to study Duchenne muscular dystrophy

PPMD, Santhera Pharmaceuticals collaborate to study Duchenne muscular dystrophy

Parent Project Muscular Dystrophy, the leading advocacy organization working to end Duchenne muscular dystrophy (Duchenne) and Santhera Pharmaceuticals, a Swiss specialty pharmaceutical company focusing on the development and marketing of innovative pharmaceutical products for the treatment of mitochondrial and neuromuscular diseases, will collaborate on a benefit/risk study in Duchenne. [More]
Cardiac stem cell treatment restores heart function damaged by Duchenne muscular dystrophy

Cardiac stem cell treatment restores heart function damaged by Duchenne muscular dystrophy

Researchers at the Cedars-Sinai Heart Institute have found that injections of cardiac stem cells might help reverse heart damage caused by Duchenne muscular dystrophy, potentially resulting in a longer life expectancy for patients with the chronic muscle-wasting disease. [More]
Murdoch University researchers receive NHMRC grant to develop genetic drugs for rare diseases

Murdoch University researchers receive NHMRC grant to develop genetic drugs for rare diseases

Murdoch University researchers from the Centre for Comparative Genomics have received almost $800,000 in funding from the National Health and Medical Research Council to develop genetic drugs to treat rare diseases. [More]
Research offers hope to patient suffering from Duchenne muscular dystrophy

Research offers hope to patient suffering from Duchenne muscular dystrophy

Research at Stockholm's KTH Royal Institute of Technology offers hope to those who suffer from Duchenne muscular dystrophy, an incurable, debilitating disease that cuts young lives short. [More]
Isoform: New protein offers novel therapeutic approach for patients with DMD

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. [More]
PTC Therapeutics initiates reimbursed expanded access program for Translarna

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

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. [More]
EMA's CHMP gives positive opinion for PTC Therapeutics' Translarna

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

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. [More]
Common drug may improve blood flow in muscles of boys with Duchenne muscular dystrophy

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. [More]
Advertisement
Advertisement