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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 makes case for gene therapy Zolgensma in SMA
Perhaps the most encouraging result from these scores is that the drug has shown potential to help those with SMA type 1 sit unassisted for at least 30 seconds. ... strategy. That came after it evaluated first-to-market SMA gene therapy Spinraza and
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Catalent preps for complex therapy boom with $1.2bn Paragon buy
And in 2018, Novartis’ bought AveXis for $8.7bn to lay claim to Zolgensma, a gene therapy for spinal muscular atrophy (SMA), while in 2016 Pfizer paid $645m for Bamboo Therapeutics.
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Biogen strikes $800m deal to buy gene therapy firm Nightstar
lay claim to a gene therapy for spinal muscular atrophy (SMA) that could threaten Biogen’s own Spinraza (nusinersen) drug treatment.
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‘Inflexible’ NICE blocking access to rare disease drugs
The most high profile example of this is Biogen’s Spinraza (nusinersen) a first-in-class treatment for Spinal Muscular Atrophy (SMA), a devastating life-limiting condition.
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Novartis aims for 10 blockbuster launches by 2020
The one-time, potentially curative treatment for spinal muscular atrophy (SMA) has attracted a great deal of interest ahead of its approval, thanks in part to Novartis saying the treatment could ... Zolgensma – spinal muscular atrophy (SMA) type 1. 2020
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How to pay for very high cost, curative drugs? ICER and NICE put their heads together
Three HTA bodies seek answers to future value models. When Novartis’ hinted last year that its SMA gene therapy Zolgensma could cost $4m per patient, it put healthcare systems on notice
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Latest Intelligence
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Drug launches to watch in 2019
The next step in this process is the launch of Zolgensma (AveXis) – the gene therapy for spinal muscular atrophy (SMA) which is now filed with the FDA and EMA, and is ... The company argues that current ten- year costs of caring for SMA type 1 patients
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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.
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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).
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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
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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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