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SMA

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

Beneluxa: the future of European market access?

Spinraza (nusinersen) is the first drug approved to treat spinal muscular atrophy (SMA), an often-fatal genetic disease, which affects muscle strength and movement, and can strike in infancy or in ... The Netherlands’ health minister Bruno Bruins said

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Better together when buying medicines? Taking stock of the BeNeLuxA initiative

SMA). The first joint negotiation to deliver a deal: Spinraza. Simple economics would predict that the bigger the buyer, the better the deal. ... There will also be additional research on the effect of Spinraza in subtypes of SMA.
Biogen picks up another drug from wide-ranging Ionis alliance

Meanwhile, Biogen recently licensed another candidate with potential in ALS from AliveGen, signing a $535m deal for rights to two myostatin inhibitors that will be tested in both ALS and SMA.
FDA fast tracks Novartis’ SMA gene therapy

Novartis is expected to provide tough competition for Biogen and its first-to-market SMA treatment, Spinraza. ... Its president David Lennon, said: “This important step by the FDA brings us ever closer to delivering Zolgensma to patients with SMA Type 1
Orphan drugs from Biogen and Amicus win UK Prix Galien

Biogen receive their award for Spinraza. In the dedicated orphan drug category, the winner was Biogen’s antisense oligonucleotide Spinraza (nusinersen), the first disease-modifying therapy for spinal muscular atrophy (SMA),
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

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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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