Tag Archives: spinal muscular atrophy

December, 2018

December, 2017

October, 2017

March, 2017

  • 17 March

    AveXis’ Gene Therapy for SMA Demonstrates Positive Results

    CHICAGO, March 16, 2017 (GLOBE NEWSWIRE) — AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today reported topline results from the Phase 1 trial of AVXS-101 in spinal muscular atrophy (SMA) Type 1. The company also reported financial …

January, 2017

December, 2016

July, 2016

  • 22 July

    FDA Grants Breakthrough Therapy Designation to AveXis’ Gene Therapy

    CHICAGO, July 20, 2016 (GLOBE NEWSWIRE) — 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 granted Breakthrough Therapy Designation for AVXS-101, the company’s lead development candidate for the …

January, 2015

  • 16 January

    Roche Expands its Rare Disease Portfolio with Trophos Acquisition

    Swiss drugmaker Roche has signed an agreement to acquire privately-held Trophos for up to $545 million. The acquisition will provide Roche with Trophos’ candidate for the rare and debilitating spinal muscular atrophy (SMA). Trophos’ proprietary screening platform generated olesoxime (TRO19622), which is in mid-stage development for SMA. SMA is a …