Tag: Dusty

Malaysian sufferers of a deadly muscle-wasting disease are denied the latest drugs

There is still no cure for a fatal but rare muscle wasting disease named after a 19th century French neurologist that affects about 300,000 boys worldwide. About 3,000 Duchenne muscular dystrophy patients are in Malaysia and as many as 300 of them are in Sabah. But new drugs developed in the last six years that hold promise of a cure have been denied them because of their prohibitive costs. Ironically, it is a Sabah woman, whose son suffers from Duchenne, who has contributed significantly to the development of the first three exon skipping drugs ever to have been approved by the American Food and Drug Administration to treat the deadly disease. More recently, the FDA has approved the first gene therapy which doctors say may be close to a cure for Duchenne. But none of the Malaysian patients, particularly those in Sabah, can avail themselves to these. A year’s treatment with Amondys 45 (casimersen), an exon skipping drug, can cost as much as RM6.5m ($1.5m) while a one-time gene therapy with Elevidys (Delandistrogene moxeparvovec) costs RM14.2m, making it one of the most expensive drugs in the world.