SMA Press
AveXis to Initiate Screening for Remaining Patients in Pivotal Trial of AVXS-101 for SMA Type 1 Following Review of Preliminary Data from First Three Patients
Chicago, Ill. (January 30, 2018) – AveXis, Inc. (NASDAQ: AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced that, following review of safety data and early signals of efficacy from the first three patients dosed in its pivotal trial of AVXS-101 for spinal muscular…
Read MoreAveXis Announces Expanded Clinical Development Program for AVXS-101in Spinal Muscular Atrophy
Chicago, Ill. (January 16, 2018)- AveXis, Inc. (NASDAQ: AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today provided an overview of the expanded clinical development program for the company’s initial gene therapy candidate, AVXS-101, for the treatment of spinal muscular atrophy (SMA). In addition to…
Read MoreAveXis Announces Plan to Initiate Phase 1 Trial in SMA Type 2 Utilizing Intrathecal Delivery of AVXS-101
Chicago, Ill. (December 13, 2017) – AveXis, Inc. (NASDAQ: AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced the U.S. Food and Drug Administration (FDA) has notified the company that, based on review of data submitted, the company may initiate its planned Phase 1…
Read MoreIonis and Biogen Win Prestigious Prix Galien Best Biotechnology Product Award for SPINRAZA
The SMA Foundation congratulates Ionis and Biogen for being awarded the Prix Galien USA Award for the Best Biotechnology Product in 2017 for SPINRAZA® (nusinersen). The Prix Galien USA Award recognizes extraordinary achievement in scientific innovation that improves the state of human health. The award was presented at a ceremony in New York City on…
Read MoreNovartis to resume enrollment in ongoing study of branaplam in type 1 SMA
Effective immediately, following the respective Health Authorities and Ethics Committee approvals, Novartis will be resuming enrollment in the ongoing study in type 1 SMA (CLMI070X2201) at their existing sites in Belgium, Germany, Denmark and Italy. Please click here to read the full letter.
Read MoreAveXis Announces Plan to Initiate Pivotal Trial of AVXS-101 in SMA Type 1 Using Product from New GMP Commercial Process
Chicago, Ill. (September 29, 2017) – AveXis, Inc. (NASDAQ: AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced the U.S. Food and Drug Administration (FDA) has notified the company that based on review of data submitted, including the potency assay, it may initiate its…
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