Dystrophin News and Research

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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.

Cardiologist receives grant to study impact of erectile dysfunction drugs on muscular dystrophy patients

A Cedars-Sinai Heart Institute cardiologist has been awarded a three-year, nearly $1 million grant from the Muscular Dystrophy Association (MDA) to study whether drugs used to treat erectile dysfunction could also be used to improve muscle blood flow and reduce fatigue in muscular dystrophy patients.

25 Mar 2010

BeaconEquity.com: AVI BioPharma's revenues for fourth-quarter of 2009 are $5.1M

BeaconEquity.com announces an investment report featuring biotech company AVI BioPharma Inc. The report includes financial, comparative and investment analyses, and pertinent industry information you need to know to make an educated investment decision.

18 Mar 2010

BioMarin Pharmaceutical reports GAAP net income of $4.7M for fourth-quarter 2009

BioMarin Pharmaceutical Inc. today announced financial results for the fourth quarter and year ended December 31, 2009. GAAP net income was $4.7 million ($0.05 per diluted share) for the fourth quarter of 2009, compared to GAAP net income of $24.5 million ($0.21 per diluted share) for the fourth quarter of 2008, which included a $30.0 million payment from Merck Serono related to the approval of Kuvan in the EU.

24 Feb 2010

Tβ4 may help treat skeletal muscle disease in Duchenne muscular dystrophy

REGENERX BIOPHARMACEUTICALS, INC. announced today that a research team in Washington, D.C. has found that dystrophin-deficient Mdx mice, treated twice a week for six months with Tβ4, showed a significant increase in skeletal muscle regenerating fibers compared to untreated mice. No effects related to muscle function or fibrosis and no adverse reactions were observed in the mice.

6 Feb 2010

PTC Therapeutics initiates additional clinical trial of ataluren in boys and young men with nmDBMD

PTC Therapeutics, Inc. (PTC) today announced the initiation of an additional clinical trial of ataluren (PTC124®) in boys and young men with nonsense mutation Duchenne/Becker muscular dystrophy (nmDBMD) who have permanently lost the ability to walk independently.

20 Jan 2010

Link between dystrophin gene and age of cardiac disease onset in BMD patients identified

Investigators in The Research Institute at Nationwide Children's Hospital have identified a link between specific modifications of the dystrophin gene and the age of cardiac disease onset in patients with Becker muscular dystrophy (BMD). This information could help clinicians provide early cardiac intervention for BMD patients based on genetic testing results performed on a blood sample. These findings are a result of analysis of the largest number of BMD patients to date and are published in the December issue of the journal Circulation: Cardiovascular Genetics.

18 Jan 2010

BioMarin Pharmaceutical commences BMN 195 Phase 1 clinical study for DMD

BioMarin Pharmaceutical Inc. announced today that the first subject has initiated treatment in the Phase 1 clinical study of BMN 195, a small molecule utrophin upregulator, for the treatment of Duchenne muscular dystrohpy (DMD). Initial top-line results are expected in the third quarter of 2010.

12 Jan 2010

AMT receives Innovation Credit to develop gene therapy treatment for DMD

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, announced today that it will receive an Innovation Credit of up to EUR 4 million from the Dutch government to support the development of AMT's gene therapy treatment for Duchenne Muscular Dystrophy (DMD).

6 Jan 2010

Humans and mice have critical differences in gene responsible for DMD

Humans and mice have previously unknown and potentially critical differences in one of the genes responsible for Duchenne muscular dystrophy (DMD). Researchers writing in the open access journal BMC Biology have found that two major features of a key DMD gene are present in most mammals, including humans, but are specifically absent in mice and rats, calling into question the use of the mouse as the principal model animal for studying DMD.

4 Dec 2009

AMT's gene therapy successful in treating Duchenne muscular dystrophy

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, announced today that it has successfully treated Duchenne muscular dystrophy (DMD) in an animal model with its proprietary gene therapy. The proof of concept studies were performed in collaboration with the group of Professor Irene Bozzoni (University of Rome, La Sapienza, Italy) and demonstrated effectiveness in the heart as well as in skeletal muscles.

11 Nov 2009

Research shows exon-skipping drug may prove effective for treating Duchenne muscular dystrophy

An exon skipping PPMO has demonstrated dramatic effects in the prevention and treatment of severely affected, dystrophin and utrophin-deficient mice, preventing severe deterioration of the treated animals and extending their lifespan.

21 Oct 2009

Amsterdam Molecular Therapeutics' AMT-080 granted EMEA Orphan Drug Designation

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, announced today that the European Medicines Agency has granted Orphan Drug Designation to AMT's gene therapy product AMT-080 for the treatment of Duchenne muscular dystrophy.

13 Oct 2009

UCLA discovery raises hope for curing genetic disorders caused by nonsense mutations

UCLA scientists have identified two chemicals that convince cells to ignore premature signals to stop producing important proteins. Published in the Sept. 28 edition of the Journal of Experimental Medicine, the findings could lead to new medications for genetic diseases, such as cancer and muscular dystrophy, that are sparked by missing proteins.

29 Sep 2009

Charley's Fund and the Nash Avery Foundation to support investigation of Galapagos' SARM candidate drug

Galapagos NV (Euronext: GLPG), Charley's Fund Inc. and the Nash Avery Foundation announced today that they will collaborate to investigate the potential effectiveness of Galapagos' SARM candidate drug, G100192, in treating Duchenne muscular dystrophy. G100192 is an orally-available small molecule therapeutic, which has demonstrated successful Proof of Concept in pre-clinical studies for cachexia (the loss of weight and muscle mass).

17 Sep 2009

AVI BioPharma's peptide-conjugated PMO chemistry to be discussed at two upcoming scientific meetings

AVI BioPharma, Inc., a developer of RNA-based drugs, today announced that Hong Moulton, Ph.D., Director of Discovery Research, will give oral presentations highlighting improved analogues of AVI's phosphorodiamidate morpholino oligomer (PMO) chemistry at two upcoming scientific meetings.

28 Aug 2009

New function for missing protein in Duchenne muscular dystrophy found

Researchers at the University of Minnesota and National Institutes of Health have identified a new function for the protein missing in people with the most common and ultimately lethal form of childhood muscular dystrophy.

3 Aug 2009

New therapy shows potential for treating Duchenne muscular dystrophy

Researchers at the University of Minnesota Medical School have discovered a new therapy that shows potential to treat people with Duchenne muscular dystrophy, a fatal disease and the most common form of muscular dystrophy in children.

26 May 2009

Inflammation clue to fragile bones in Duchenne's muscular dystrophy

Inflammation could contribute to bone loss in Duchenne's muscular dystrophy (DMD), a discovery made by a group of Italian researchers. Dr Anna Rufo and her colleagues found that levels of an inflammatory molecule, known as IL-6, are high in patients with DMD.

25 May 2009

First treatment for muscular dystrophy a step closer

Genetic researchers at Children's National Medical Center and the National Center of Neurology and Psychiatry in Tokyo published the results of the first successful application of "multiple exon-skipping" to curb the devastating effects of Duchenne muscular dystrophy in an animal larger than a mouse.

16 Mar 2009

Novel genetic technology for canine form of muscular dystrophy

Using a novel genetic technology that covers up genetic errors, researchers funded in part by the National Institutes of Health have developed a successful treatment for dogs with the canine version of Duchenne muscular dystrophy, a paralyzing, and ultimately fatal, muscle disease.