Clinical Trials

The clinical research program at the INMR focuses on developing accurate measurement tools and identifying new treatments for inherited and acquired nerve and muscle disorders of childhood.

To reach this aim a number of clinical trials and long term studies of natural history are being conducted with people who volunteer to take part.  Carefully conducted clinical trials are the fastest and safest way to find treatments that work to improve health.

Each study answers specific scientific questions and tries to find better ways to prevent, screen for, diagnose, or treat a disease.

Current research studies at the INMR include:

Peripheral Neuropathy
  Australasian Paediatric Charcot-Marie-Tooth disease Registry
•  Investigation of the neuronal types of Charcot-Marie-Tooth disease in children
•  Characterizing the peripheral neuropathy in children with mitochondrial disease
•  Identifying the genetic basis of inherited peripheral neuropathies in children
•  Rehabilitation, surgical and pharmacological therapies for Charcot-Marie-Tooth disease

Muscular Dystrophy
•  Quality of life in boys with Duchenne Muscular Dystrophy
•  New steroid and other treatments for Duchenne muscular dystrophy
•  Exon skipping for Duchenne muscular dystrophy
•  Disease modifying genes in muscle disease
These studies are being undertaken at CHW singly or in collaboration with internationally-based clinical trials groups including the Cooperative International Neuromuscular Research Group (CINRG), which is based in Washington DC, Inherited Neuropathies Consortium, TREAT-NMD and the European Neuromuscular Centre neuromuscular research consortium.