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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.

Novartis buys gene therapy firm AveXis for $8.7bn

Novartis buys gene therapy firm AveXis for $8.7bn

The Swiss pharma group is paying $218 per share in cash for the US biotech and its lead gene therapy candidate for spinal muscular atrophy (SMA), with the price tag more

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  • 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.

  • 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

  • Deal Watch August 2016 Deal Watch August 2016

    95. Ionis (US). Biogen (US). Exercise option . Nusinersen, in phase III for treatment for spinal muscular atrophy.

  • 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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  • 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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