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spinal muscular atrophy

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

Astellas makes gene therapy a core focus with $3bn Audentes buy

Astellas makes gene therapy a core focus with $3bn Audentes buy

For instance, the $2.1m Novartis charges for its spinal muscular atrophy therapy  Zolgensma, now the most expensive drug in the world, helped the therapy to $160m in sales in its ... Meanwhile, Audentes has several other candidates in development,

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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).

  • 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

  • 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

  • The good, the bad and the ugly The good, the bad and the ugly

    Tecfidera (MS) and Spinraza (Spinal Muscular Atrophy) are expected to generate big sales, while Tysabril (MS and Crohn’s Disease) and Avonex (MS) are expected to see sales drop due to

  • Europe vs the US: New drug product approvals Europe vs the US: New drug product approvals

    Among the noteworthy orphan approvals are Exondys 51 (for the treatment of Duchenne muscular dystrophy, only approved in the US), Darzalex (for the treatment of multiple myeloma, approved in the US ... 2015 and in the EU 2016) and Spinraza (for the

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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

  • Raising Awareness: Rare Disease Day

    of, such as spinal muscular atrophy and lysosomal storage disorders.(3). ... confused for other diseases.(2) For example, the genetic disorder adrenomyeloneuropathy, characterized by progressive problems with the brain, spinal cord and peripheral nerves

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