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).

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

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

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

Duchenne Muscular Dystrophy News and Research

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

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

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

Sensing a Healthier Future

Achieving and maintaining ISO/IEC 27001 and BS 10012 certifications

Expanding research and clinical options for children with cancer

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