trial

/Tag: trial

FSHD Research Projects

Advertising and promoting Facioscapulohumeral dystrophy (FSHD) Research Projects Researchers at The Royal Children’s Hospital are looking for children and adolescents aged 6-18 years who have been diagnosed with FSHD, to participate in one or both of these studies: The effect of the nutritional supplement (creatine monohydrate) on strength and muscle mass in children with FSHD [...]

By |2018-09-14T09:40:25+00:00September 14th, 2018|disorders, research, trials|0 Comments

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+00:00April 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+00:00November 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+00:00October 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+00:00July 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+00:00July 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+00:00July 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+00:00July 21st, 2016|Comments Off on Spinal Muscular Atrophy An Overview