Peter McWilliams

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biotechnology research replacement therapies rare diseases pmm2-cdg glycosylation clinical development glm101 orphan drugs therapeutics enzyme deficiency mannose-1-phosphate disease-modifying treatment genetic mutation protein dysfunction biochemical pathways clinical trials u.s. fda u.s. ema sugar molecule delivery substrate replacement protein misfolding therapy pipeline clinical benefit patient quality of life genetic testing metabolism disorders n-glycosylation protein structure immune system regulation protein interactions developmental delays cognitive impairment treatment options ataxia phase 2 study investigational drug natural history study orphan disease rare pediatric disease risk factors glycan function enzyme activity clinical efficacy molecular therapy health solutions therapeutic candidates patient care medical need biotechnology company drug development scientific advancements health care health wellness & fitness hospital & health care