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).

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

'Molecular band-aid' may help protect hearts of Duchenne muscular dystrophy patients

Researchers at the University of Minnesota Medical School have discovered a chemical that may, over the long term, protect the hearts of Duchenne muscular dystrophy patients - a fatal and most common form of muscular dystrophy in children.

16 Mar 2010

CureDuchenne: Scientific advisors to come together to find cure for DMD

Today, CureDuchenne, a nonprofit dedicated to raising funds and awareness for Duchenne Muscular Dystrophy (DMD), announced that on March 19, 2010 the organization’s scientific advisors from around the country will come together in the fight to find a cure for Duchenne Muscular Dystrophy.

4 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

AMT announces full year 2009 results; reports modest decrease in operating expenses

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today reported its results for the year to December 31, 2009.

24 Feb 2010

Genzyme announces fourth-quarter and full-year 2009 financial results

Genzyme Corp., a diversified, global biotechnology company, today announced fourth-quarter and full year 2009 financial results and provided 2010 guidance that reflects growth across its businesses and a focus on strengthening core areas of the company.

18 Feb 2010

Special issue reviews current state of science in fetal therapy

Repairing birth defects in the womb. Inserting a tiny laser into the mother's uterus to seal off an abnormal blood flow and save fetal twins. Advancing the science that may allow doctors to deliver cells or DNA to treat sickle cell anemia and other genetic diseases before birth.

17 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

BioMarin announces update on BMN 110 Phase I/II trial for Morquio A Syndrome

BioMarin Pharmaceutical Inc. today announced an update on the Phase I/II trial for BMN 110 or N-acetylgalactosamine 6-sulfatase (GALNS), intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), or Morquio A Syndrome. Preliminary clinical data from the first 24 weeks of the study (12 weeks at 0.1mg/kg and 12 weeks at 1.0 mg/kg) have been evaluated, and BioMarin plans to announce top-line results for the full 36-week study after completion of dosing at 2.0 mg/kg in the second quarter of 2010.

5 Feb 2010

BioMarin Pharmaceutical signs stock purchase agreement to acquire LEAD Therapeutics

BioMarin Pharmaceutical Inc. announced today that it has entered into a stock purchase agreement to acquire LEAD Therapeutics, Inc. (LEAD), a small private drug discovery and early stage development company with key compound LT-673, an orally available poly (ADP-ribose) polymerase (PARP) inhibitor for the treatment of patients with rare, genetically defined cancers.

4 Feb 2010

A novel approach for funding children's genetic disease research

Parent Project Muscular Dystrophy together with Trisomy 18 Foundation are among 100 charities that earned a $25,000 grant during Round 1 of the Chase Community Giving Challenge on Facebook.

19 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

Kennedy Krieger Institute opens new center for individuals with muscle disorders

Kennedy Krieger Institute announced today the opening of the new Center for Genetic Muscle Disorders that will improve and expand services to individuals with muscle disorders such as muscular dystrophy and congenital myopathy in the Baltimore/Washington region and across the nation. The Center provides expert clinical care to children and adults with these disorders, and offers leading research programs both in the clinical and laboratory setting.

9 Dec 2009

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

Amsterdam Molecular Therapeutics provides third-quarter 2009 business updates

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today provides its non-audited business update in compliance with the EU transparency directive. This report summarizes material events and AMT's financial position for the third quarter of 2009.

18 Nov 2009

Galapagos third quarter 2009 financial results in line with management expectations

Galapagos NV announces today that third quarter 2009 operational and financial results were in line with management expectations. These results encourage the Company to retain full year guidance of EUR 100 million revenues, a growth of 30% compared to 2008. Galapagos' service division BioFocus will significantly increase its cash contribution to the Group result for the full year 2009.

13 Nov 2009

Gene therapy increases muscle size and strength with no adverse effects in monkeys: MDA

Four macaque monkeys that received injections of genes for a protein called follistatin into upper leg muscles experienced pronounced and durable increases in muscle size and strength and no adverse effects, the Muscular Dystrophy Association (MDA) announced today. The findings could have implications particularly for injured and aging people worldwide; and for tens of millions experiencing muscle loss associated with cancer, AIDs and muscle diseases.

12 Nov 2009

Gene delivery strategy to help improve muscle mass in patients with degenerative muscle disorders

Severe weakness of the quadriceps is a defining feature of several neuromuscular disorders. Researchers at Nationwide Children's Hospital have shown that a gene delivery strategy that produces follistatin - a naturally occurring protein that inhibits myostatin, a growth factor expressed specifically in skeletal muscle - directly to the quadriceps of non-human primates results in long-term gene expression with muscle enhancing effects, including larger muscles with greater strength.