Tuesday 26 April 2011

Update: Partial Clinical Hold Lifted by FDA for GSK2402968 (Exon 51 Skip)

Update: Partial Clinical Hold Lifted by FDA for GSK2402968 (Exon 51 Skip): "GlaxoSmithKline (GSK) has provided an update regarding the clinical development program for GSK2402968 (exon 51 skip) for DMD: The US Food and Drug Administration has lifted the partial clinical hold on the Investigational New Drug Application for GSK2402968. This means GSK can now proceed with longer term clinical studies of GSK2402968 in DMD patients in the US."

ACE-031 statement from Acceleron and Shire - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

ACE-031 statement from Acceleron and Shire - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: "ACE-031 STATEMENT FROM ACCELERON AND SHIRE
Clinical trial suspended; intention is to start a new study after discussions with regulatory agencies"

Tuesday 12 April 2011

Parent Project Muscular Dystrophy: GSK 2402968 (PRO-051)

Parent Project Muscular Dystrophy: GSK 2402968 (PRO-051): "the forty-eight week follow-up data from the phase I/IIa extension study (PRO051-02) will be presented at an oral session on April 13, 2011 during the 63rd annual meeting of the American Academy of Neurology."

Run for Scotland - DeanWidd - Stopwasting - Action Duchenne - Fighting for a cure for muscular dystrophy

Run for Scotland - DeanWidd - Stopwasting - Action Duchenne - Fighting for a cure for muscular dystrophy: "If you are a runner rather than a walker then why not get involved with the 5k fun run around Holyrood Park Edinburgh on Sunday 5th June.

Starting at Holyrood Park at 9.30am runners of all abilities are welcome to get involved and help raise awareness and funds for Action Duchenne. All runners will receive a medal , completion certificate and a goody bag."

AVI BioPharma Receives Approval of Nonproprietary Name Eteplirsen for Lead Duchenne Muscular Dystrophy Therapeutic Candidate, AVI-4658 - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

AVI BioPharma Receives Approval of Nonproprietary Name Eteplirsen for Lead Duchenne Muscular Dystrophy Therapeutic Candidate, AVI-4658 - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: "AVI BioPharma (NASDAQ: AVII), a developer of RNA-based therapeutics, today announced that the United States Adopted Names (USAN) Council has approved the nonproprietary name eteplirsen for AVI-4658, the Company's exon-skipping therapy for the treatment of Duchenne muscular dystrophy (DMD), a genetic muscle wasting disease caused by the absence of functional dystrophin. In addition, the World Health Organization (WHO) has approved eteplirsen as the International Nonproprietary Name (INN) for AVI-4658."

Golden Mile 1,2 or 3 mile walk - DeanWidd - Stopwasting - Action Duchenne - Fighting for a cure for muscular dystrophy

Golden Mile 1,2 or 3 mile walk - DeanWidd - Stopwasting - Action Duchenne - Fighting for a cure for muscular dystrophy: "Starting at 11:30am on Sunday 5th June 2011, the Golden Mile Walks are primarily aimed at all who wish to participate in what will be a tremendous fun day out. Whether you wish to walk or stroll 1,2, or 3 miles the choice is yours.Anybody can take part and no one is excluded"

Parent Project Muscular Dystrophy: Home

Parent Project Muscular Dystrophy: Home: "Once again, TRND is accepting proposals, due April 26, 2011. This program is part of a congressionally mandated program to encourage and speed the development of new drugs for rare and neglected diseases. This unique program creates a drug development pipeline within the NIH and is specifically intended to stimulate research collaborations with academic scientists, non-profit organizations, and pharmaceutical and biotechnology companies working on rare and neglected illnesses."

Friday 8 April 2011

Wednesday 6 April 2011

Action Duchenne welcomes backtrack on NHS plans - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Action Duchenne welcomes backtrack on NHS plans - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: "ACTION DUCHENNE WELCOMES BACKTRACK ON NHS PLANS
Charity states that government plans that threaten the most vulnerable must now be re-examined, young men to lobby parliament for access to treatment"

Tuesday 5 April 2011

Phrixus Pharmaceuticals, Inc. Announces $890,000 in NIH Funding for Its Programs in Duchenne Muscular Dystrophy and Heart Failure

Phrixus Pharmaceuticals, Inc. Announces $890,000 in NIH Funding for Its Programs in Duchenne Muscular Dystrophy and Heart Failure: "Phrixus Pharmaceuticals, Inc., a clinical-stage, specialty pharmaceutical company focused on innovative therapies for Duchenne muscular dystrophy (DMD) and heart failure, today announced that it has received a total of $890,000 in awards from the National Institutes of Health (NIH). Funding is in the form of one SBIR Phase 1 award titled 'Effects of P-188 on Respiratory Function and Diaphragm Degeneration in the mdx Mouse' and one STTR Phase 1 award titled 'Poloxamer 188 Mechanism of Action in Ischemic Heart Failure.' The latter award is in collaboration with Dr. Joseph M. Metzger, Chair of Integrative Biology and Physiology at the University of Minnesota."

Cedars-Sinai receives $750,000 grant from Parent Project Muscular Dystrophy for sildenafil/taladafil study

Cedars-Sinai receives $750,000 grant from Parent Project Muscular Dystrophy for sildenafil/taladafil study: "Cedars-Sinai receives $750,000 grant from Parent Project Muscular Dystrophy for sildenafil/taladafil study"

Parent Project Muscular Dystrophy: Cialis® (Tadalafil)

Parent Project Muscular Dystrophy: Cialis® (Tadalafil): "In Duchenne (and some cases of Becker) the lack of dystrophin causes, among other things, a decrease in nitric oxide, which normally stimulates cGMP and holds blood vessels open during muscle contraction. Without adequate NO levels, it is thought that the muscles of those with Duchenne and Becker may experience “ischemia,” or damage due to a lack of oxygen. By blocking PDE-5, tadalafil may be able to compensate for the loss of NO at the muscle membrane by upregulating cGMP directly and protecting the muscles of those with Duchenne/Becker during exercise."

Friday 1 April 2011

Google Reader (10)

Google Reader (10): "Mitch Sevier, a talented photographer and father of a son with Duchenne, has put together some incredible images that we feel truly capture what Duchenne “looks” like. These photographs illustrate Duchenne in a way PPMD has never experienced."