Wall Street Journal |
Gene Therapy, New Drug Fight Rare Disease in Kids
WebMD WEDNESDAY, Nov. 1, 2017 (HealthDay News) -- Babies born with a previously untreatable degenerative nerve disease now have two fresh sources of hope for their future. Two innovative new therapies for spinal muscular atrophy (SMA) type 1 have proven ... Two New Ways to Treat A Deadly Disease: Spinal Muscular Atrophy Gene-replacement therapy helping kids with neuromuscular disease The Most Expensive Drug in the World Is About to Be Made Worthless by Gene Therapy |
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