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

2016APBD

Jan 1

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

MDBR2016APBD

Million Dollar Bike Ride

The efficacy of guaifenesin in the treatment of adult polyglucosan body disease

A multi-center, prospective, minimal intensity BMT trial for dyskeratosis congenita