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  • Research and Markets: 2015 Funded Research in Spinal Muscular Atrophy
    Research and Markets has announced the addition of the "Funded Research in Spinal Muscular Atrophy" report to th

  • Isis Pharmaceuticals Earns $7M for Advancing ISIS-SMN Rx in Children With Spinal Muscular Atrophy
    ISIS-SMNRx is designed to alter the splicing of a closely related gene (SMN2) to increase production of fully functional SMN protein.  The FDA granted orphan drug status and fast track designation to ISIS-SMNRx for the treatment of patients with SMA.  Isis is currently in collaboration with Biogen Idec to develop and potentially commercialize the investigational compound, ISIS-SMNRx, to treat ...

  • Mechanistic insights into spinal muscular atrophy suggest new paths for treatment
    ( Cold Spring Harbor Laboratory ) Today, a team of researchers from Cold Spring Harbor Laboratory sheds new light on the underlying pathology of spinal muscular atrophy, a rare but devastating disease that causes muscle weakness and paralysis and is the leading genetic cause of infant deaths. With no approved drugs currently available, the newly obtained insights may prove valuable as scientists ...

  • Anthony Rizzo: Cubs will win NL Central
    Chicago Cubs players Mike Olt, left, and Anthony Rizzo, center, visit with 10-month-old Mia Gallardo and her mom, Elizabeth Villa Gallardo, at Advocate Lutheran General Hospital in Park Ridge on Thursday. Baby Mia and her twin sister, Maya, both have spinal muscular atrophy (SMA) and have...

  • Kay Insurance Services Continues Its Community Improvement Efforts in San Antonio, TX by Introducing a Charity ...
    Kay Insurance Service Inc. begins a new charity campaign in San Antonio, Texas as an alleged Agents of Change Mission. The new highlighted community cause campaign is helping to raise funds to provide financial relief to a 10-month-old infant recently diagnosed with Spinal Muscular Atrophy, and his family. (PRWeb January 24, 2015) Read the full story at ...

  • Isis Pharmaceuticals Earns Milestone Payment from Biogen - Analyst Blog
    Isis Pharmaceuticals (ISIS) earned a milestone payment of $7 million from partner Biogen for the advancement of the ongoing open-label extension study on ISIS-SMNRx in children with spinal muscular atrophy.

  • Paralysed Coventry girl waves at classmates for first time
    Jaime Sale, who has spinal muscular atrophy, has had a new arm support fitted to help her regain some movement

  • Roche buys France's Trophos
    Looking to gain access to an experimental drug to treat spinal muscular atrophy, Roche ( OTCQX:RHHBY ) has agreed to buy privately-held Trophos.

  • Kacie Martin: Mum's heartfelt appeal to improve dying 'angel' daughter's last days
    Mum Nicole Williams has taken to the internet to try and buy a wheelchair for her dying little girl. Loving Nicole, 21, and dad Shaun, 25, are begging people to give just a pound so two-year-old daughter Kacie can enjoy what is left of her life.

  • Severely disabled Baroness Campbell's speech against Assisted Dying Bill
    Severely disabled crossbench peer Baroness Campbell of Surbiton, who suffers from severe spinal muscular atrophy, reveals her fears over plans to give some terminally ill people the right to die.

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