January 16, 2018
AveXis, 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...
January 9, 2018
Scholar Rock, a biotechnology company based in Boston, has announced that they will be investing nearly $50 million to bring SRK-105, a muscle drug for possible treatment of SMA, into human clinical trials. SRK-105 is Scholar Rock’s
January 5, 2018
Working together, we’ve achieved tremendous success in advocating for ourselves, our families, and our whole community, particularly in the past few years.
Today, we’re building on this momentum with the launch of our