The neuromuscular clinical team within the neurology department at PCH are excited to have joined an international clinical trial offering hope of a breakthrough treatment for families of boys with Duchenne Muscular Dystrophy (DMD), a rare form of muscular dystrophy.
The trial will test the effectiveness of a new drug ‘Ataluren’ targeting a specific genetic mutation in patients with DMD, a devastating disease which not only causes significant disability but also a shortened life expectancy.
DMD is a neuromuscular disorder caused by mutations in the DMD gene that mostly affects boys. There are presently over 45 boys under the age of 18 who have been diagnosed with DMD in WA.
Neurologist and principle investigator on the trial Dr Maina Kava said there was strong interest in this trial among families affected because until now there was no treatment or trials that could be offered to these patients in WA.
“While this trial is limited to DMD patients with a specific genetic mutation, it represents hope for all DMD patients that it will help pave the way towards potential treatment options for all in the future.
“Our research team has worked hard to establish this trial at PCH because we are passionate about providing the best possible care to our patients with this debilitating disease,” said Dr Kava. Read more on HealthPoint.
Pictured | (l-r) Neurologist Dr Maina Kava, Clinical nurse Jodi Mann with Mitchell and Aiden Hillyard.