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Latest Duchenne muscular dystrophy Stories

2010-02-24 00:51:00

AMSTERDAM, February 24, 2010 /PRNewswire-FirstCall/ -- Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, today reported its results for the year to December 31, 2009. Highlights - Glybera(R) Marketing Authorisation Application submitted to European Medicines Agency (EMA, formerly known as EMEA); - EMA commenced formal review of Glybera(R) dossier on January 20, 2010; - EMA grants orphan drug designation for AMT's...

2010-01-21 16:56:51

A study published in today's online edition the American Journal of Human Genetics, allowed the first identification of a new form of adult onset muscular dystrophy. The research team led by Dr. Bernard Brais, neurogeneticist at the Research Centre of the Centre hospitalier de l'Universit© de Montr©al (CRCHUM) and associate professor, Universit© de Montr©al, in collaboration with European collaborators, demonstrated that recessive ANO5 mutations will lead to abnormal...

2010-01-19 13:09:00

Dedicated to Saving Children's Lives in CHASE COMMUNITY GIVING CHALLENGE MIDDLETOWN, Ohio and DALE CITY, Va., Jan. 19 /PRNewswire-USNewswire/ -- 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. Now in Round 2, Chase Bank has given them the opportunity to compete for an additional $1 Million prize and five $100,000 prizes, to be decided by public...

2010-01-19 08:00:00

SOUTH PLAINFIELD, N.J., Jan. 19 /PRNewswire/ -- 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. This trial is evaluating the best methods for measuring functional abilities in patients who have lost independent mobility. Patients with nmDBMD develop...

2010-01-14 14:45:48

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...

2010-01-11 08:00:00

NOVATO, Calif., Jan. 11 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) 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. "Duchenne muscular dystrophy represents a serious unmet medical need affecting approximately 40,000 patients in the developed world,...

2010-01-06 01:20:00

AMSTERDAM, January 6 /PRNewswire-FirstCall/ -- Amsterdam Molecular Therapeutics (Euronext: AMT), 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). The credit is granted by SenterNovem, an agency of the Dutch Ministry of Economic affairs. "We are delighted to receive this credit for DMD,...

2010-01-05 11:00:00

New Additions to Organization Will Focus on Research, Advocacy, and Outreach MIDDLETOWN, Ohio, Jan. 5 /PRNewswire-USNewswire/ -- Patricia A. Furlong, Founding President and CEO of Parent Project Muscular Dystrophy (PPMD), the largest non-profit organization in the United States focused on finding a cure for Duchenne muscular dystrophy (Duchenne), announced today that Sharon Hesterlee, Ph.D. and Holly Peay, MS, CGC, will be joining the PPMD staff. Dr. Hesterlee joins PPMD as Senior...

2009-12-08 07:00:00

SOUTH PLAINFIELD, N.J., Dec. 8 /PRNewswire/ -- Data published online in the medical journal Muscle and Nerve support the use of the six-minute walk test (6MWT) as an outcome measure in PTC Therapeutics, Inc.'s ongoing registration-directed clinical trial of ataluren in patients with nonsense mutation Duchenne/Becker muscular dystrophy (nmDMD/BMD). The study results, which will also be published in an upcoming print issue of the journal, characterize the limitations on walking by patients...

2009-12-04 14:17:53

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. Roland Roberts led a team of researchers from King\'s...


Word of the Day
holluschickie
  • A 'bachelor seal'; a young male seal which is prevented from mating by its herd's older males (mated bulls defending their territory).
This comes from the Russian word for 'bachelors.'
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