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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

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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  • Drug launches to watch in 2019 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

  • No incentive for a cure 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 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 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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