hereditary spinal ataxia

(redirected from Friedreichs ataxia)
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hereditary spinal ataxia

[hə′red·ə‚ter·ē ¦spīn·əl ə′tak·sē·ə]
(medicine)
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Bamboos portfolio includes potential best-in-class rAAV-based gene therapies that will complement Pfizers rare disease and gene therapy portfolios in two priority areas: neuromuscular, with a pre-clinical asset for Duchenne Muscular Dystrophy (DMD); and central nervous system, with pre-clinical assets for Friedreichs Ataxia and Canavan disease, and a Phase I asset for Giant Axonal Neuropathy.
Then he gave Mark - who had been in a wheelchair since the age of 10 with Friedreichs Ataxia - his mum Mary and sister Caroline his limo to drive back to their hotel.
Upon completion of the proposed acquisition, the combined companys pipeline will consist of Avalanches existing ophthalmic programs and four new gene therapy based programs, which are focused on Alpha1-antitrypsin (A1AT) deficiency, cardiomyopathy associated with Friedreichs ataxia, hereditary angioedema and severe allergies.