We will be posting a series of summaries from our 2015 researcher meeting, highlighting some of the most interesting new developments and discoveries presented there. This update covers a session on clinical research and outcome measures. The session was moderated by Thomas Crawford, MD. Clinical Research Studies and Outcome Measures This session started off with […]
2015 SMA Researcher Meeting Summary: Clinical Research Read More »
We will be posting a series of summaries from our 2015 researcher meeting, highlighting some of the most interesting new developments and discoveries presented there. This update covers a session on newborn screening. The session was moderated by Kathryn Swoboda, MD. Newborn Screening for SMA: Ethics, Rationale, and Implementation Data generated in mouse models of
2015 SMA Researcher Meeting Summary: Newborn Screening Read More »
At the Annual SMA Conference, representatives from six of the seven programs currently in clinical trials gave an update on their programs. Of these six programs, four—gene therapy, ISIS-SMNRx, RG7800, and LMI070—treat the underlying genetics of SMA. Gene therapy aims to correct the mutation in the SMN1 gene that causes SMA, and the other three
Updates from Drug Programs in Clinical Trials Read More »
We will be posting a series of summaries from our 2015 researcher meeting, highlighting some of the most interesting new developments and discoveries presented there. This update covers the special session, “Emerging Trends in Motor Neuron Pathobiology.” This summary was written by Cure SMA Scientific Advisory Board member Doug Kerr, MD, PhD. Emerging Trends in
2015 SMA Researcher Meeting Summary: Emerging Trends in Motor Neuron Pathobiology Read More »
The House of Representatives has passed 21st Century Cures. Cure SMA is proud to support this legislation. Thank you for your calls and tweets to voice your support to your Representatives. They really made a difference! So what happens next? The Senate is busy drafting their version of the bill, called “Innovation,” and we expect
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