SMA is an often-deadly genetic neuromuscular disorder caused by a missing or defective gene that normally produces a protein needed for development of motor neurons in the spinal cord
A DRUG being co-developed by Roche to treat spinal muscular atrophy (SMA) helped improve development scores in babies with the genetic disease, a study released showed, as the race heats up for therapies destined to be among the drug industry’s most expensive.
PTC Therapeutics, which struck a licensing deal with Roche in 2011 for its SMA programme, said more than 90 percent of babies with severe Type 1 SMA given the RG7916 drug achieved a greater than fourpoint increase in a test to measure their neuromuscular progress six months after treatment began.
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