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Isis Initiates Phase 1 Clinical Study of ISIS-SMNRx in Patients with Spinal Muscular Atrophy
…a devastating disease that leads to the loss of motor neurons resulting in muscle weakness and respiratory failure in children. The genetic cause of this disease is well understood, but…
Read MoreRoche Signs Agreement with PTC Therapeutics to Advance Treatment for Spinal Muscular Atrophy (SMA)
…protein. The compounds in PTC’s research treat the underlying cause of the disorder and demonstrate increases in SMN levels in nervous system, muscles and other tissues in SMA models. SMA…
Read MoreRoche Signs Agreement with PTC Therapeutics to Advance Treatment for Spinal Muscular Atrophy (SMA)
…protein. The compounds in PTC’s research treat the underlying cause of the disorder and demonstrate increases in SMN levels in nervous system, muscles and other tissues in SMA models. SMA…
Read MoreIsis Pharmaceuticals Initiates Phase 3 Study of ISIS-SMNRX in Infants with Spinal Muscular Atrophy
…For further study information, please visit www.clinicaltrials.gov and search for ISIS-SMNRx or the identifier number NCT02193074 or visit the ISIS-SMNRx study site at www.smastudy.com. ABOUT ISIS-SMNRx ISIS-SMNRx is designed…
Read MoreOrally delivered compounds, which selectively modify RNA splicing, prevent deficits in mouse models of Spinal Muscular Atrophy
…important step towards developing a potential therapeutic option for this devastating and currently untreatable condition. Early clinical trials are currently underway to determine the safety and tolerability of this approach.”…
Read MoreBiogen Idec and Isis Pharmaceuticals Announce Global Collaboration for Antisense Program Targeting Spinal Muscular Atrophy
Biogen Idec and Isis Pharmaceuticals Announce Global Collaboration for Antisense Program Targeting Spinal Muscular Atrophy — Biogen Idec has Option to Develop and Commercialize Promising Compound for Most Common Genetic…
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