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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.

Orphan drugs from Biogen and Amicus win UK Prix Galien

Orphan drugs from Biogen and Amicus win UK Prix Galien

Biogen receive their award for Spinraza. In the dedicated orphan drug category, the winner was Biogen’s antisense oligonucleotide Spinraza (nusinersen), the first disease-modifying therapy for spinal muscular atrophy (SMA),

Latest news

  • Novartis: pipeline will generate 60 filings in three years Novartis: pipeline will generate 60 filings in three years

    Already active in this area with its lymphoma and leukaemia CAR-T therapy Kymriah (tisagenlecleucel), Novartis is highlighting new projects today including gene therapy AVXS101 for spinal muscular atrophy (SMA) type

  • Spinraza lifts Biogen ahead of Alzheimer’s data Spinraza lifts Biogen ahead of Alzheimer’s data

    Spinraza lifts Biogen ahead of Alzheimer’ s data. Continued strong growth for Biogen’s spinal muscular atrophy therapy Spinraza helped the company exceed revenue expectations in the third quarter, but pipeline

  • Novartis buys cancer specialist Endocyte for $2.1bn

    The latest deal comes shortly after Novartis agreed a $8.7bn deal to acquire AveXis, a US specialist in spinal muscular atrophy (SMA).

  • Roche drug hits the mark in spinal muscular atrophy studies Roche drug hits the mark in spinal muscular atrophy studies

    Roche drug hits the mark in spinal muscular atrophy studies. Data suggests Risdiplam could improve motor function. ... Roche has positive clinical data on its risdiplam candidate in spinal muscular atrophy (SMA), suggesting it could add to the emerging

  • Pfizer abandons antibody for Duchenne Pfizer abandons antibody for Duchenne

    Pfizer abandons antibody for Duchenne. Pfizer has halted development of its Duchenne muscular dystrophy (DMD) candidate domagrozumab on disappointing results in a mid-stage efficacy trial. ... Biogen is however focusing initially on spinal muscular

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Latest Intelligence

  • 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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