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A Primer for Family Medicine and Primary Care: Focusing on Pompe Disease
Recognizing Pompe disease, a progressive, debilitating and often fatal neuromuscular disease caused by a genetic deficiency or dysfunction of the lysosomal enzyme acid alpha-glucosidase (GAA), can be challenging, as its signs and symptoms are like those of other...
POMPE Survey
A biotech company that conducts research and develops treatments for people living with rare diseases such as Pompe disease wants your help. They are recruiting Pompe patients and caregivers to take a short online survey about your knowledge and understanding of...
Amicus Therapeutics Announces Regulatory and Clinical Updates for AT-GAA in Pompe Disease
Amicus Therapeutics Announces Regulatory and Clinical Updates for AT-GAA in Pompe Disease CRANBURY, N.J., Sept. 10, 2018 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), a global biotechnology company focused on discovering, developing and delivering novel...
CLINICAL TRIALS/STUDIES
A FEW OF THE CLINICAL TRIALS/STUDIES THAT ARE CURRENTLY RECRUITING. FOR A COMPLETE LIST VISIT WWW.CLINICALTRIALS.GOV
Higher Lumizyme Dose May Help Prevent Pompe Progression in Infants, Case Report Suggests
Higher Lumizyme Dose May Help Prevent Pompe Progression in Infants, Case Report Suggests JULY 18, 2018BY ALICE MELAOIN NEWS. Treatment with Lumizyme(alglucosidase alfa) at double the approved dose may help prevent the rapid decline of respiratory...
Amicus Therapeutics Strengthens Biologics Business Strategy
Amicus Therapeutics Strengthens Biologics Business Strategy
Valerion Therapeutics Demonstrates a New Mechanism for Treating Pompe Disease
PR NewsWire March 3, 2017 Valerion Therapeutics Demonstrates a New Mechanism for Treating Pompe Disease Concord, MA-- March 3, 2017-- Valerion Therapeutics announced today that it has developed a fusion protein, VAL-1221, which combines its proprietary antibody...
Amicus Therapeutics Announces Positive Preliminary Data from Phase 1/2 Study of Novel Treatment Paradigm for Pompe Disease
December 8, 2016 Amicus Therapeutics Announces Positive Preliminary Data from Phase 1/2 Study of Novel Treatment Paradigm for Pompe Disease ATB200/AT2221 Safety Data Show No Infusion-Associated Reactions Following 100+ Infusions Clinical Pharmacokinetic (PK) Profile...
Sanofi Genzyme Begins Pivotal Phase 3 Trial of NeoGAA Investigational Second-Generation Therapy for Pompe Disease
PRESS RELEASE Sanofi Genzyme Begins Pivotal Phase 3 Trial of NeoGAA Investigational Second-Generation Therapy for Pompe Disease Cambridge, Mass. – November 4, 2016 - Sanofi Genzyme, the specialty care global business unit of Sanofi, announced today that the first...
Guidance to the Pompe Community
Guidance to the Pompe Community