Duchenne Muscular Dystrophy News and Research

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Duchenne muscular dystrophy (DMD) is a progressive muscle disorder that causes the loss of both muscle function and independence. DMD is perhaps the most prevalent of the muscular dystrophies and is the most common lethal genetic disorder diagnosed during childhood today. Each year, approximately 20,000 children worldwide are born with DMD (one of every 3,500 male children).

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

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

Race to Yes campaign lauds Sarepta's plans to seek FDA approval for Duchenne treatment

The Race to Yes campaign today lauded drug maker Sarepta on its plans to move forward immediately to seek FDA approval of the first drug to successfully treat Duchenne muscular dystrophy, the world's leading genetic killer of children.

22 Apr 2014

Sarepta Therapeutics plans to submit eteplirsen NDA for treatment of Duchenne muscular dystrophy

Sarepta Therapeutics, Inc., a developer of innovative RNA-based therapeutics, today announced it plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration by the end of 2014 for the approval of eteplirsen for the treatment of Duchenne muscular dystrophy (DMD).

21 Apr 2014

Research suggests that person can slow aging process by exercising regularly

New research by Canadian sports medicine physician Mark Tarnopolsky, MD, PhD, suggests that a person can slow the speed at which they age by exercising regularly. Dr. Tarnopolsky presented his research titled, "Exercise as a Countermeasure for Aging: From Mice to Humans" today at the 23rd Annual Meeting of the American Medical Society for Sports Medicine (AMSSM). Dr. Tarnopolsky discussed how regular exercise not only improves the quality of life but can also extend a person's lifespan by up to five years.

8 Apr 2014

Thomson Reuters, Children's Tumor Foundation partner to create neurofibromatosis pathway maps

The Intellectual Property & Science business of Thomson Reuters, the world's leading provider of intelligent information for businesses and professionals, today announced an expansion of its partnership with the Children's Tumor Foundation to create unique neurofibromatosis (NF) pathway maps aimed at significantly increasing the understanding of the disease and its underlying mechanisms.

4 Apr 2014

Device to improve blood flow implanted in muscular dystrophy patient

"Today, we're going to make history," said 18-year-old Eric Ramos on the day UT Southwestern Medical Center doctors operated on his ailing heart.

29 Mar 2014

Takeda works with UCL to drive research into tackling muscle disorders

Japanese pharmaceutical company Takeda will work with University College London (UCL) to drive research into tackling muscle disorders, in particular muscular dystrophy.

10 Mar 2014

Sarepta Therapeutics reports non-GAAP net loss of $29.1 million for Q4 2013

Sarepta Therapeutics, Inc., a developer of innovative RNA-based therapeutics, today reported financial results for the three months and year ended December 31, 2013, and provided an update of recent corporate developments.

27 Feb 2014

Researchers demonstrate new approach to treating muscular dystrophy

Researchers at Washington University School of Medicine in St. Louis have demonstrated a new approach to treating muscular dystrophy. Mice with a form of this muscle-weakening disease showed improved strength and heart function when treated with nanoparticles loaded with rapamycin, an immunosuppressive drug recently found to improve recycling of cellular waste.

12 Feb 2014

New approach for reducing levels of toxic protein fragments associated with Huntington's disease

An innovative therapeutic strategy for reducing the levels of toxic protein fragments associated with Huntington's disease uses a new approach called exon skipping to remove the disease-causing component of the essential protein, huntingtin.

11 Feb 2014

Scientists develop new potential cure for rare eye disease

University of British Columbia and Vancouver Coastal Health scientists have developed a potential cure for a rare eye disease, showing for the first time that a drug can repair a birth defect.

22 Dec 2013

Retrophin to acquire privately-held company, Kyalin Biosciences

Retrophin, Inc. today announced that it will acquire privately-held Kyalin Biosciences, Inc., an early-stage company based in San Diego, CA, that is developing therapies targeting the core symptoms of autism and related conditions.

12 Dec 2013

Retrophin signs agreement with Novartis for exclusive U.S. license for Syntocinon Nasal Spray

Retrophin, Inc. today announced that it has signed an agreement with Novartis Pharma AG for an exclusive U.S. license for Syntocinon Nasal Spray, the intranasal formulation of a synthetic version of the naturally occurring peptide hormone oxytocin, for an upfront payment of $5.0 million plus milestone payments and royalties.

12 Dec 2013

Tackling orphan diseases: an interview with Damian Marron, CEO, TxCell

The exact definition of orphan diseases varies depending on where you are in the world. In the EU it's defined as a prevalence of less than 1 in 2,000, which equates to about 250,000 patients across the EU.

4 Dec 2013

New zebrafish technique finds potential treatments for multiple diseases

Skeletal muscle has proved to be very difficult to grow in patients with muscular dystrophy and other disorders that degrade and weaken muscle. Researchers at Boston Children's Hospital's Stem Cell Program now report boosting muscle mass and reversing disease in a mouse model of Duchenne muscular dystrophy, using a "cocktail" of three compounds identified through a new rapid culture system. Adding the same compounds to stem cells derived from patients' skin cells, they then successfully grew human muscle cells in a dish.

8 Nov 2013

Researchers overcome obstacle to using viruses to deliver therapeutic genes

Scientists in The Research Institute at Nationwide Children's Hospital have found a way to overcome one of the biggest obstacles to using viruses to deliver therapeutic genes: how to keep the immune system from neutralizing the virus before it can deliver its genetic payload. In a study published recently in Molecular Therapy, researchers found that giving subjects a treatment to temporarily rid the body of antibodies provides the virus safe passage to targeted cells, allowing it to release a corrective or replacement gene to treat disease.

30 Oct 2013

New type of antibiotic kills multidrug-resistant germ common to health care settings

A new type of antibiotic called a PPMO, which works by blocking genes essential for bacterial reproduction, successfully killed a multidrug-resistant germ common to health care settings, UT Southwestern Medical Center researchers report.

16 Oct 2013

Duchenne Muscular Dystrophy News and Research

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

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

Race to Yes campaign lauds Sarepta's plans to seek FDA approval for Duchenne treatment

Sarepta Therapeutics plans to submit eteplirsen NDA for treatment of Duchenne muscular dystrophy

Research suggests that person can slow aging process by exercising regularly

Thomson Reuters, Children's Tumor Foundation partner to create neurofibromatosis pathway maps

Device to improve blood flow implanted in muscular dystrophy patient

Takeda works with UCL to drive research into tackling muscle disorders

Sarepta Therapeutics reports non-GAAP net loss of $29.1 million for Q4 2013

Researchers demonstrate new approach to treating muscular dystrophy

New approach for reducing levels of toxic protein fragments associated with Huntington's disease

Scientists develop new potential cure for rare eye disease

Retrophin to acquire privately-held company, Kyalin Biosciences

Retrophin signs agreement with Novartis for exclusive U.S. license for Syntocinon Nasal Spray

Tackling orphan diseases: an interview with Damian Marron, CEO, TxCell

New zebrafish technique finds potential treatments for multiple diseases

Researchers overcome obstacle to using viruses to deliver therapeutic genes

New type of antibiotic kills multidrug-resistant germ common to health care settings

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