Amicus Therapeutics Announces First Patient Dosed in Phase 3 PROPEL Pivotal Study of AT-GAA in Patients with Pompe DiseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More Global Study to Assess AT-GAA Compared to Standard-of-Care Enzyme Replacement Therapy (ERT)A treatment that replaces the missing enzyme through IV infusion. More over 52 Weeks CRANBURY, N.J., Dec. 20, 2018— Amicus...

Spark Therapeutics Announces New Preclinical Data for Pompe DiseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More Gene TherapyA developing treatment that aims to fix, replace, or add genetic instructions. More Candidate Data from IND-enabling studies in three species support moving SPK-3006 into the clinic in 2019 PHILADELPHIA, Oct. 08, 2018—Spark Therapeutics, a fully integrated, commercial...

SPARK THERAPEUTICS GENE THERAPYA developing treatment that aims to fix, replace, or add genetic instructions. More RESEARCH WEBINAR Please join the AMDA for a webinar focused on Spark Therapeutics and gene therapyA developing treatment that aims to fix, replace, or add genetic instructions. More research Take this opportunity to learn more about Spark as a company and the field of gene therapyA developing treatment that aims to fix, replace, or add genetic instructions. More research. We will be joined by...

The AMDA is excited to announce a research grant opportunity for applicants who are involved in Pompe DiseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More research. This grant opportunity was made possible by the 2018 Pull for Pompe, a private fundraiser held by the Precast Manufacturer’s Association of Texas...

CRANBURY, N.J., Sept. 10, 2018—Amicus Therapeutics, a global biotechnology company focused on discovering, developing and delivering novel medicines for rare metabolic diseases, announced today regulatory and clinical advancements in its development program AT-GAA for...

Researchers from the Erasmus MC in Rotterdam, Netherlands, have succeeded in generating muscle cells from skin cells. This is significant as skin cells can be relatively easily obtained from a skin biopsy. The team developed a protocol to obtain pure muscle cells (ie...

The AMDA is excited to announce a research grant opportunity for applicants who are involved in Pompe DiseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More research. This grant opportunity was made possible by the 2017 Pull for Pompe, a private fundraiser held by the Precast Manufacturer’s Association of Texas...

CONCORD, Mass., July 11, 2017—Valerion Therapeutics, a clinical-stage biotechnology company that specializes in the development of therapies for orphan genetic diseases, today announced that it has initiated dosing in a Phase 1/2 clinical trialA research study that tests new treatments or approaches in people. More evaluating VAL-1221 in...

he AMDA was saddened to learn of the sudden passing of Henri Termeer, former CEO of Genzyme. We have known and worked with Henri since the late-90s through his role as the CEO of Genzyme. We will never forget how he always took the time to talk to patients and their...

Excerpt . . . . CRANBURY, N.J., May 15, 2017 —Amicus Therapeutics today announced positive functional data from initial patients in a global Phase 1/2 study (ATB200-02) to investigate ATB200/AT2221 in patients with Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More. Patients who completed six months of...