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Research and Markets: Spinal Muscular Atrophy (SMA) Therapeutics Pipeline Review 2014 - 17 Companies & 27 Drug Profiles
[September 12, 2014]

Research and Markets: Spinal Muscular Atrophy (SMA) Therapeutics Pipeline Review 2014 - 17 Companies & 27 Drug Profiles


DUBLIN --(Business Wire)--

Research and Markets (http://www.researchandmarkets.com/research/n86r4p/spinal_muscular) has announced the addition of the "Spinal Muscular Atrophy (SMA) - Pipeline Review, H2 2014" report to their offering.

This report provides comprehensive information on the therapeutic development for Spinal Muscular Atrophy (SMA (News - Alert)), complete with comparative analysis at various stages, therapeutics assessment by drug target, mechanism of action (MoA), route of administration (RoA) and molecule type, along with latest updates, and featured news and press releases. It also reviews key players involved in the therapeutic development for Spinal Muscular Atrophy (SMA) and special features on late-stage and discontinued projects.

The report enhances decision making capabilities and help to create effective counter strategies to gain competitive advantage. It strengthens R&D pipelines by identiying new targets and MOAs to produce first-in-class and best-in-class products.



Companies Involved in Therapeutics Development

  • Genzyme Corporation
  • F. Hoffmann-La Roche Ltd.
  • Isis Pharmaceuticals, Inc.
  • Pfizer Inc.
  • Zambon Company S.p.A.
  • Paratek Pharmaceuticals, Inc.
  • Cytokinetics, Inc.
  • Neurotune AG
  • Trophos SA
  • Nexgenix Pharmaceuticals, LLC
  • Genethon
  • Vybion, Inc.
  • OrphageniX, Inc.
  • Retrophin, LLC
  • Neurodyn Inc.
  • GMP-Orphan SAS (News - Alert)
  • Cellular Biomedicine Group, Inc.

Drug Profiles


  • olesoxime
  • ISIS-SMNRx
  • PF-06687859
  • RG-7800
  • Gene Therapy to Activate SMN for Spinal Muscular Atrophy
  • AAV9-SMN Gene Therapy
  • tirasemtiv
  • PTK-SMA-2
  • NXD-30001
  • DNA Oligonucleotides for Spinal Muscular Atrophy
  • RE-003
  • Gene Therapy for Spinal Muscular Atrophy
  • ND-602
  • Antisense Oligonucleotide for Spinal Muscular Atrophy and Myotonic Dystrophy
  • Motor Neuron Precursor Cells
  • Small Molecule to Activate SMN Protein for Spinal Muscular Atrophy
  • Agrin Biologic For SMA
  • sodium phenylbutyrate
  • Small Molecules for CNS Disorders
  • PMO-25
  • Synthetic Peptides for Central Nervous System Disorders
  • BBrm-01
  • Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy
  • Oligonucleotide to Activate SMN2 for Spinal Muscular Atrophy
  • INT-41
  • Antisense Oligonucleotides for Spinal Muscular Atrophy
  • ZP-037

For more information visit http://www.researchandmarkets.com/research/n86r4p/spinal_muscular


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