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SMA

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

Novartis: pipeline will generate 60 filings in three years

Already active in this area with its lymphoma and leukaemia CAR-T therapy Kymriah (tisagenlecleucel), Novartis is highlighting new projects today including gene therapy AVXS101 for spinal muscular atrophy (SMA) type

Latest news

Novartis buys cancer specialist Endocyte for $2.1bn

The latest deal comes shortly after Novartis agreed a $8.7bn deal to acquire AveXis, a US specialist in spinal muscular atrophy (SMA).
Roche drug hits the mark in spinal muscular atrophy studies

in untreated type 1 SMA – and the same number also achieved upright head control. ... Roche is also moving ahead with two more studies to further explore how risdiplam might be used in SMA.
Pfizer abandons antibody for Duchenne

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

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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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