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SMA

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

Roche scores FDA priority review for risdiplam in SMA

Roche scores FDA priority review for risdiplam in SMA

In addition to the SUNFISH study of type 2 and type 3 SMA patients, Roche is currently investigating risdiplam in the FIREFISH clinical trial in infants with type 1 SMA. ... If Roche scores approval for the drug, it will compete with Biogen’s

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  • Orphan drugs and breakthrough therapies drive Cortellis Drugs to Watch 2019 Orphan drugs and breakthrough therapies drive Cortellis Drugs to Watch 2019

    the genetic defect underlying spinal muscular atrophy (SMA). ... In the third quarter of 2018, AveXis filed Zolgensma for approval in the US, EU and Japan for the treatment of SMA type I.

  • Bluebird: on a mission to ‘recode’ the DNA of healthcare Bluebird: on a mission to ‘recode’ the DNA of healthcare

    Bluebird is not the only company likely to break through this 1m barrier in 2019 – Novartis’ SMA gene therapy Zolgensma is also on the cusp of approval in the US.

  • 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

    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  the health of motor

  • 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).

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  • Article: Market access in Brazil

    All eyes on Spinraza. Director Rachel Howard asks, “What does Brazil’s unprecedented risk-sharing agreement in Spinal Muscular Atrophy (SMA) mean for the future of market access in Brazil? ... In April 2019, Brazilian Health Minister Luiz Henrique

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