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Search Results - Spinal Muscular Atrophy
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  • Biogen Idec Pays Isis $3.5M for Antisense Drug Milestone
    Idec associated with the dosing of the first patient in a Phase II study of ISIS-SMN Rx in infants with spinal muscular atrophy (SMA) as part of a deal inked in January of 2012 . ...
    5-10-2013
  • Repligen, Pfizer Make $70M Spinal Muscular Atrophy Deal
    Repligen and Pfizer entered into a licensing agreement to advance Repligen's spinal muscular atrophy (SMA) program, originally in-licensed from Families of SMA, a patient ...
    1-3-2013
  • Isis Gets More Money from Biogen Idec
    $29 million up front for a deal centered on development and commercialization of Isis' Phase I-stage antisense candidate for the treatment of spinal muscular atrophy, ISIS-SMN RX . ...
    12-10-2012
  • The 10 Most Well-Connected People in Biotech
    ... (MedIC) Coalition Friends of Cancer Research, Executive Committee and member Spinal Muscular Atrophy Foundation, Board of Directors Columbia University Medical Center, Board of ...
    10-29-2012
  • Funds Awarded for MS Drug Clinical Trial
    The drug is currently undergoing Phase II evaluation for the treatment of spinal muscular atrophy, and has separately demonstrated promising results in models of Huntington's ...
    10-11-2012
  • Target Discovered for ALS
    ... expression of another mutant protein, TDP-43, and also blocked the motor axon abnormalities caused by deletion of Smn1, a zebrafish model used to study spinal muscular atrophy. ...
    8-27-2012
  • BioEurope Spring Day 3
    to announce a deal worth 6.7 million Euros from the patient group AFM. The finance will enable them to do a full efficacy study for their lead compound in spinal muscular atrophy. ...
    8-13-2012
  • Synthetic Biology Firms Ally on Infectious Disease mAbs
    collaboration with BioLife Cell Bank, focused on leveraging Intrexon's technologies to develop autologous, genetically modified stem cell therapeutics for spinal muscular atrophy. ...
    8-8-2012
  • Biogen Idec Pays Isis $12M in Antisense Drug Deal
    worldwide option and collaboration agreement focused on the development of Isis' antisense investigational drug, ISIS-SMN Rx , for the treatment of spinal muscular atrophy (SMA). ...
    6-29-2012
  • Expression Analysis Stakes Claim on RNA-Seq
    ... including the International Serious Adverse Events Consortium, the Spinal Muscular Atrophy Foundation, the Cure Huntington's Disease Foundation, and International ...
    6-15-2012
  • With Oligonucleotide Drugs, Antisense Arrives but RNAi Has a...
    In January Biogen Idec paid $29 million up front for an option to Isis' ISIS-SMNRx program to treat spinal muscular atrophy (SMA). Biogen also agreed to up to $45 million in ...
    6-8-2012
  • Retrophin Takes on Ligand's Nephropathy Candidate DARA for $1M...
    Retrophin is developing a pipeline of fusion protein candidates for the potential treatment of Duchenne muscular dystrophy (DMD), spinal muscular atrophy, cystic fibrosis, and ...
    2-21-2012
  • Biogen Idec Explains Company's Move Away from Venture Funding
    ... And with Isis Pharmaceuticals, Biogen Idec announced January 4 an option to license Isis' antisense drug ISIS-SMN Rx , a treatment for spinal muscular atrophy. It's a matter of ...
    1-27-2012
  • Biogen Idec Pays Isis $29M for Option to Early-Clinical SMA Antisense...
    collaboration agreement centered on development and commercialization of the latter's Phase I-stage antisense candidate for the treatment of spinal muscular atrophy, ISIS-SMN RX . ...
    1-4-2012
  • Roche Nabs PTC's SMA Program for $30M Up Front
    Roche is paying PTC Therapeutics $30 million up front for an exclusive, worldwide license to the latter's spinal muscular atrophy (SMA) program. It includes three preclinical-stage ...
    11-29-2011
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    Journal Articles

  • A Model of Motor Neuron Loss: Selective Deficits after Ricin...
    Margaret Coutts, Ling X. Kong, Hans S. Keirstead
    Journal of Neurotrauma
    motor system models lower limb paralysis and the deficits that occur in diseases like amyotrophic lateral sclerosis (ALS) and infantile progressive spinal muscular atrophy (SMA). ...
  • Decreasing Disease Severity in Symptomatic, Smn-/- ; SMN2+/+...
    Jacqueline J. Glascock, Erkan Y. Osman, Mary J. Wetz, Megan M. Krogman, Monir Shababi, Christian L. Lorson
    Human Gene Therapy and Part B: Methods
    Decreasing Disease Severity in Symptomatic, Smn-/- ; SMN2+/+ , Spinal Muscular Atrophy Mice Following scAAV9-SMN Delivery Human Gene Therapy and Part B: Methods Abstract Spinal ...
  • Trans -Splicing, More Than Meets the Eye: Multifaceted Therapeutics...
    Matthew E.R. Butchbach
    Human Gene Therapy and Part B: Methods
    Trans -Splicing, More Than Meets the Eye: Multifaceted Therapeutics for Spinal Muscular Atrophy Human Gene Therapy and Part B: Methods
  • In Vitro Restoration of Functional SMN Protein in Human Trophoblast...
    Federica Sangiuolo, Antonio Filareto, Paola Spitalieri, Maria Lucia Scaldaferri, Ruggiero Mango, Emanuela Bruscia, Gennaro Citro, Ercole Brunetti, Massimo De Felici, Giuseppe Novelli
    Human Gene Therapy and Part B: Methods
    In Vitro Restoration of Functional SMN Protein in Human Trophoblast Cells Affected by Spinal Muscular Atrophy by Small Fragment Homologous Replacement Human Gene Therapy and Part ...

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