Injecting Wnt7a protein could prevent Duchenne muscular dystrophy

Injecting Wnt7a protein could prevent Duchenne muscular dystrophy: Scientists have discovered that injecting a novel human protein into muscle affected by Duchenne muscular dystrophy significantly increases its size and strength, findings that could lead to a therapy akin to the use of insulin by diabetics. These results were published today in the Proceedings of the National Academy of Sciences by Dr. Julia von Maltzahn and Dr. Michael Rudnicki, the Ottawa scientist who discovered muscle stem cells in adults.

Great news from Holyrood

Great news from the debate results from Holyrood

The meeting last night at Holyrood was a great success. The Public
Health Minister, Michael Matheson, advised that £16 million will be
allocated for wheelchair services, £1.5 million for respiratory
services and finally although no specific amount was stated he said
funding will be given into research for Duchenne! This is a fantastic
result which has been a long time coming!

John Miller, grandparent to Lee, a great friend of Action Duchenne's
worked tirelessly to get this debate to happen and we are very
grateful for his wonderful efforts.

Summit Corporation PLC Press Release - Repeat Dosing of SMT C1100 for DMD meets Endpoints in phase 1 trial - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Summit Corporation PLC Press Release - Repeat Dosing of SMT C1100 for DMD meets Endpoints in phase 1 trial - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: Oxford, UK, 7 November 2012 - Summit (AIM: SUMM), a UK drug discovery company,
announces that the repeat dosing of the utrophin upregulator SMT C1100 for the
treatment of the fatal muscle-wasting disease Duchenne Muscular Dystrophy
('DMD') has successfully met the endpoints as part of a Phase 1 clinical trial
in healthy volunteers. The trial evaluated a new formulation of SMT C1100 and
the results showed that upon repeat dosing, concentrations of the drug achieved
in the blood plasma, stabilised at levels that from preclinical studies are
expected to significantly increase utrophin protein production. The new
formulation was also shown to be safe and well-tolerated in this Phase 1 trial.

HCT 1026 may help stem debilitating effects of muscular dystrophy

HCT 1026 may help stem debilitating effects of muscular dystrophy: Cedars-Sinai Heart Institute researchers have found that an experimental compound may help stem the debilitating effects of muscular dystrophy by restoring normal blood flow to muscles affected by the genetic disorder.

The researchers studied HCT 1026, a new type of molecule in which nitric oxide is chemically attached to a standard anti-inflammatory agent, in a preclinical model of muscular dystrophy. Results suggest HCT 1026 may be beneficial for the treatment of Duchenne muscular dystrophy, which begins in early childhood, and Becker muscular dystrophy, which often occurs later in adulthood.

New Ataluren UK Clinical Trials - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

New Ataluren UK Clinical Trials - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: We are delighted to inform you that PTC Therapeutics has been able to establish an extension study in the UK for their drug ataluren for patients who participated in the Phase 2b. This extension study will take place at two centres: Newcastle and London. The background to this decision by PTC Therapeutics can be found at http://www.actionduchenne.org/viewarticle?news=387 .
Secondly, PTC Therapeutics is in active communication with European regulators to run a wider confirmatory study with the aim of confirming the findings of the previous study. We look forward to welcoming PTC Therapeutics to our forthcoming annual conference where we hope there will be an opportunity to find out more about their plans.

First approval for AAV vector gene therapy - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

First approval for AAV vector gene therapy - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: Netherlands biotech company UniQure have announced that Glybera has become the first gene therapy approved by regulatory authorities in the Western world.

It is the first medication approved for patients with rare metabolic disorder Lipoprotein Lipase Deficiency and commercial roll-out to begin second half of 2013. The company that has taken this product over from AMT state that it now validates uniQure’s unique AAV-based gene therapy platform.