Experts and patient organisations unite to discuss DMD clinical trial parameters - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

On the 21st of June, Diana Ribeiro, Head of Research, Action Duchenne was involved in a meeting hosted by TREAT-NMD and co-sponsored by various MD organisations entitled Ways to Measure Clinical Effectiveness in the Investigation of Medicinal Products for BMD/DMD. This was a document drafted by the European Medicines Agency earlier this year (link below) and has gone out to consultation to all. They have invited further evidence-based comments and feedback in a coherent and structured manner by the 31st of August. 

The meeting led by the experts and family members pointed out areas they felt had not been clear with regards to current progress in assessing DMD clinical trials and based on current knowledge can be addressed further. A few representatives from the regulatory agencies were present and shared their personal views. Much of the discussion involved the robustness and validity of current and future outcomes for studies. 

There is more evidence to emerge for clinical trial criteria, which is currently unpublished to address some of the main limitations as stipulated by the respective agencies and regulators in the draft guideline document. These need to be assimilated by the experts into a formal response and the information from this meeting and consultation will be shared with all as soon as these become available.

Updates in exon skipping data - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Updates in exon skipping data - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: Sarepta Therapeutics, Inc. announced updated data at the Wells Fargo Securities Healthcare Conference on the 19th of June from Study 202, a Phase IIb open-label extension study of eteplirsen for patients who have a genotype amenable to skipping of exon 51. Results at 84 weeks showed a continued stabilisation of walking ability in eteplirsen-treated patients evaluable on the 6-minute walk test (6MWT).As previously reported, increased novel dystrophin was observed as assessed by muscle biopsy at week 48 and is now in the long-term extension phase in which patients continue to be followed for safety and clinical outcomes. 

Duke researchers find novel way to repair gene responsible for Duchenne muscular dystrophy

Duke researchers find novel way to repair gene responsible for Duchenne muscular dystrophy: Using a novel genetic 'editing' technique, Duke University biomedical engineers have been able to repair a defect responsible for one of the most common inherited disorders, Duchenne muscular dystrophy, in cell samples from Duchenne patients.

Instead of the common gene therapy approach of adding new genetic material to "override" the faulty gene, the Duke scientists have developed a way to change the existing mutated gene responsible for the disorder into a normally functioning gene.The Duke researchers believe their approach could be safer and more stable than current methods of gene therapy.

Scottish Lobby 2013 - Action Duchenne - Fighting for a cure for muscular dystrophy

Scottish Lobby 2013 - Action Duchenne - Fighting for a cure for muscular dystrophy: SCOTTISH LOBBY 2013: ACTION DUCHENNE: BREAKTHROUGH TREATMENTS FOR DUCHENNE MUSCULAR DYSTROPHY EVENT

Please join us at Action Duchenne's Scottish Lobby event on Thursday 13th June from 1-2.30pm at the Scottish Parliament in Committee Room 1.

ARMGO Pharma gets $1 million to support ARM210 preclinical work for DMD treatment

ARMGO Pharma gets $1 million to support ARM210 preclinical work for DMD treatment: ARMGO Pharma and the Muscular Dystrophy Association (MDA) today announced that $1 Million has been awarded for preclinical work in support of an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) for ARM210, a novel, orally available, small-molecule Rycal drug that has potential as a treatment for Duchenne muscular dystrophy (DMD). The funding award to ARMGO Pharma comes from MDA Venture Philanthropy (MVP), part of MDA's translational research program.

The project being advanced by ARMGO Pharma is focused on a novel class of small molecule drugs known as Rycals™ which target the Ryanodine Receptor (RyR), an intracellular calcium channel that becomes leaky in disease states including muscular dystrophy, contributing to loss of muscle strength and function.� Rycals have been shown in animal models of muscle disease to repair RyR-mediated calcium leak and thereby improve specific muscle force and exercise capacity.�

Glaxo Muscular Dystrophy Drug Reaches Primary Objective in Study - Bloomberg

Glaxo Muscular Dystrophy Drug Reaches Primary Objective in Study - Bloomberg: GlaxoSmithKline Plc (GSK)’s drisapersen achieved the primary objective of a mid-stage study, heating up competition with Sarepta Therapeutics Inc. (SRPT) in the race for a drug that could reverse the debilitating effects of Duchenne muscular dystrophy.
Patients taking drisapersen showed a clinically meaningful difference from those on placebo after 48 weeks of treatment, according to an abstract of the study results posted on the website of CureDuchenne, an investor in Glaxo’s partner on the drug, Dutch biotechnology company Prosensa Therapeutics BV. Glaxo will present the data at a genetics conference in Cold Spring Harbor, New York, tomorrow.

Positive updates for exon skipping and other treatments - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Positive updates for exon skipping and other treatments - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: At a recent RNA and Oligonucleotide Therapeutics conference at Cold Spring Harbor, US, 10-13 April 2013, Dr John Kraus GSK Project Physician Leader presented Phase II results. The (DMD114117) study was an exploratory, unpowered, double blind, placebo-controlled clinical trial with the aim of assessing the safety and efficacy of drisapersen in 53 boys with DMD. The dosing regimens were continuous (6mg/kg/wk) and intermittent (10-week cycles of 9 doses at 6mg/kg over 6 wks, and 4 wks off drug).