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Biogen Submits NDA to the FDA for Nusinersen
Biogen and Ionis have taken a crucial step in the pathway to approval of nusinersen. Biogen has completed the submission of a New Drug Application (NDA) to the Food and…
Read MoreThe New York Times: “When Not Knowing a Celebrity Could Prove Fatal”, by Adam Cohen
…access to sitcom stars over those who don’t. Congress and the N.I.H should make the medical bureaucracy more open to patient groups, and they should do a better job of…
Read MoreAveXis Receives U.S. FDA Breakthrough Therapy Designation for AVXS-101 Gene Replacement Therapy for SMA Type 1
…has requested the company submit a Type B meeting request for a multidisciplinary, comprehensive discussion of the development program for AVXS-101. The company plans to submit the meeting request later…
Read MorePartnership between SMA Foundation, PTC Therapeutics, and Roche featured in article
Following the approval of Evrysdi, Roche released an article describing its partnership with the SMA Foundation and PTC Therapeutics to develop the drug. To read the article, please visit: https://www.roche.com/partnering/spinal-muscular-atrophy-sma-together.htm…
Read MoreScholar Rock Announces Publication of Preclinical Data on the Therapeutic Benefit of Inhibiting Myostatin Activation in Models of Spinal Muscular Atrophy
…The publication can be accessed at: http://www.scholarrock.com/platform/publications/ Long, K., O’Shea, K., Khairallah, R., et al. Specific Inhibition of Myostatin Activation is Beneficial in Mouse Models of SMA Therapy. 2018. Human…
Read MoreSMA Foundation and BG Medicine Announce Collaboration
…or call (646) 253-7100. About BG Medicine BG Medicine is a life sciences company focused on the discovery, development and commercialization of novel molecular diagnostics based on biomarkers to improve…
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