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SMA

This page shows the latest SMA news and features for those working in and with pharma, biotech and healthcare.

Pfizer abandons antibody for Duchenne

Biogen is however focusing initially on spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS).

Latest news

Shock as NICE turns down Biogen’s SMA therapy

Shock as NICE turns down Biogen’ s SMA therapy. UK’s cost effectiveness watchdog says the drug’s long-term benefits are unclear. ... in the UK with all other types of SMA do not have access to the drug.
Spinraza keeps Biogen buoyant ahead of Alzheimer’s data

There was some deceleration in new patients starting treatment with the drug, but Biogen maintains that expanding use in adult SMA patients as well as children will unlock a “large underserved ... Biogen chief executive Michel Vounatsos responded by
Novartis buys gene therapy firm AveXis for $8.7bn

Last year, Biogen secured approval for the first drug treatment for SMA, a progressive, debilitating muscle-wasting disease that affects about 23, 500 patients worldwide. ... Only around 8% of SMA patients from a matched, historic cohort were still alive
NICE finally starts review of Biogen’s Spinraza for SMA

NICE finally starts review of Biogen’ s Spinraza for SMA. Will evaluate the treatment based on its Single Technology Appraisal process. ... In trials, Spinraza has been show to improve survival in infants with SMA and reduce the chances that they go on
Biogen wins European spinal muscular atrophy first

Biogen wins European spinal muscular atrophy first. Spinraza wins accelerated approval to treat patients with the most common form of SMA. ... mechanical ventilation procedures, and showing that its benefits extend to children with less-severe,

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Latest Intelligence

No incentive for a cure

to 12-week course. And Spinraza from Biogen, the first ever disease-modifying treatment for spinal muscular atrophy (SMA), which increases the body’s ability to produce SMN protein critical to.
Deal Watch July 2016

In 2014 the partners expanded the agreement for CK-2127107 to include some neuromuscular indications, such as Spinal Muscular Atrophy (SMA).
Pharma deals during January 2013

Repligen announced the signature of an exclusive worldwide licensing agreement with Pfizer to advance Repligen's spinal muscular atrophy (SMA) programme. ... SMA is an orphan neurodegenerative genetic disease that presents early in life.

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Latest appointments

Warp Drive Bio names Gregory Verdine as CEO

He has also served as a global consultant for Roche, scientific adviser to Vertex and Ariad, and currently sits on the boards of the US National Cancer Institute and the SMA

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