Treatment of adult polyglucosan body disease caused by an intronic mutation in glycogen branching enzyme gene.

2016APBD
Written By Million Dollar Bike Ride

Awardee: Akman Hasan

Institution: Columbia University Medical Center

Award Amount: $50,500

Funding Period: January 1, 2017 - December 31, 2017

Million Dollar Bike Ride
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The efficacy of guaifenesin in the treatment of adult polyglucosan body disease
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A multi-center, prospective, minimal intensity BMT trial for dyskeratosis congenita