trial

/Tag: trial

FDA Approves Drug for Treatment of DMD

Chantal Coles PhD NMDRC provides a Research Update... On September 19th last year the U.S Food and Drug Administration (FDA) approved the first drug for the treatment of Duchenne Muscular Dystrophy, called EXONDYS 51™. This drug, formally known as Eteplirsen, is a gene modifying agent that bypasses a stop codon of the dystrophin, forming a [...]

By | 2018-06-01T03:49:09+00:00 April 3rd, 2017|information, research, trials|0 Comments

Spinal Muscular Atrophy (SMA) Research Update

Spinal Muscular Atrophy (SMA) Research update and treatment - AUSTRALIA A research update by Prof. Monique Ryan Director, Department of Neurology Group Leader, Murdoch Children's Research Institute,  Royal Children's Hospital There has been a lot of activity in the clinical trial space for SMA in the last two years. Two hospitals in Australia- the The [...]

By | 2018-06-01T03:49:11+00:00 November 17th, 2016|atrophies, research, trials|0 Comments

Muscle Clinic

Muscle Clinic A world-first initiative, the MDA Neuromuscular Clinic is funded by MDA (we have invested in excess of $1.5 million) is an initiative work alongside Neurologists and their team of specialist, all forming part of the medical team monitoring and caring for our clients. We work in conjunction with RCH team to provide the [...]

By | 2018-07-06T16:44:27+00:00 October 24th, 2016|Comments Off on Muscle Clinic

Clinical trial of new steroid for DMD

Clinical trial of new steroid for DMD ReveraGen BioPharma has announced the start of a Phase 1 clinical trial of a new steroidal drug, VBP15, for Duchenne muscular dystrophy (DMD). It is hoped that VBP15 will have at least the same benefits as the currently used corticosteroids, but fewer side effects. The trial, being conducted [...]

By | 2018-07-02T14:35:23+00:00 July 23rd, 2016|Comments Off on Clinical trial of new steroid for DMD

Clinical trial results for Oculopharyngeal MD

Clinical trial results for Oculopharyngeal MD The results of a clinical trial have shown that transplanting cells from unaffected muscles into the throat may help with the swallowing difficulties that affect people with oculopharyngeal muscular dystrophy (OPMD). OPMD is a rare type of muscular dystrophy which is unusual because it primarily affects two small muscle [...]

By | 2018-07-02T14:37:12+00:00 July 23rd, 2016|Comments Off on Clinical trial results for Oculopharyngeal MD

Sarepta apply for approval of Duchenne MD drug

Sarepta apply for approval of Duchenne MD drug Sarepta Therapeutics has issued a press release which gives an update on their plans for the development of exon skipping drug “eteplirsen”. Eteplirsen is a ‘molecular patch’ designed skip a portion of the dystrophin gene called ‘exon 51’. It has the potential to treat around 13 percent [...]

By | 2018-07-03T15:27:53+00:00 July 23rd, 2016|Comments Off on Sarepta apply for approval of Duchenne MD drug

Spinal Muscular Atrophy An Overview

What Is Spinal Muscular Atrophy? Spinal muscular atrophy (SMA) is a genetic condition which affects the nerves that control muscle movement– the motor neurons. It is named 'spinal' because most the motor neurons are located in the spinal cord. 'Muscular' is in the name because it primary affects the muscles which don’t receive signals from [...]

By | 2018-06-01T03:48:56+00:00 July 21st, 2016|Comments Off on Spinal Muscular Atrophy An Overview

MDA’s Sponsorship of CINRG

MDA's Sponsorship of CINRG  The Cooperative International Neuromuscular Research Group, (CINRG), was formed in 1999 as the clinical research arm of the Duchenne Muscular Dystrophy Research Center (DMDRC) and the Research Center for Genetic Medicine at the Children’s National Medical Center (CNMC). The primary goal of the CINRG research program is to provide a continuum [...]

By | 2018-06-01T03:49:31+00:00 September 27th, 2007|disorders, research|0 Comments