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Orally delivered compounds, which selectively modify RNA splicing, prevent deficits in mouse models of Spinal Muscular Atrophy
…and development of orally administered, proprietary small molecule drugs that target post-transcriptional control processes. Post-transcriptional control processes regulate the rate and timing of protein production and are essential to proper…
Read MoreTreatment Extends Survival in Mouse Model of Spinal Muscular Atrophy.
…While the normal form of SMN1 produces a full-length functional protein, most of the protein produced by SMN2 is truncated and unable to function. The relatively small amount of normal…
Read MoreRoche Signs Agreement with PTC Therapeutics to Advance Treatment for Spinal Muscular Atrophy (SMA)
…Therapeutics, Inc. PTC is a biopharmaceutical company focused on the discovery, development and commercialization of orally administered small-molecule drugs that target post-transcriptional control processes. Post-transcriptional control processes regulate the rate…
Read MoreRoche Signs Agreement with PTC Therapeutics to Advance Treatment for Spinal Muscular Atrophy (SMA)
…Therapeutics, Inc. PTC is a biopharmaceutical company focused on the discovery, development and commercialization of orally administered small-molecule drugs that target post-transcriptional control processes. Post-transcriptional control processes regulate the rate…
Read MoreTerms of Use
…provide the services requested by the SMA Foundation. You expressly consent to the sharing of your personal information with our service providers for the purpose of providing such services. …
Read MorePatient Advisory Board
…and Chief Scientific Officer of SMART (Spinal Muscular Atrophy Research Team – www.smarthope.com), a not-for-profit organization started in 2009 that funds novel, direct-to-investigator research aimed at finding a cure for…
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