Amicus Therapeutics Announces Presentations and Posters at the 17th Annual WORLDSymposium 2021

CRANBURY, N.J., Jan. 21, 2021 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), a patient-dedicated global biotechnology company focused on discovering, developing and delivering novel medicines for rare diseases, today announced that three oral presentations and ten posters highlighting its development programs for Lysosomal Disorders will be included at the 17th Annual WORLDSymposium 2021, to be held virtually February 8-12, 2021.

Oral Platform Presentations:

• Direct intercellular cross-correction of α-galactosidase-A deficiency in Fabry disease podocytes through tunneling nanotubes in a mixed cell culture model – Behzad Najafian, MD, Department of Pathology, University of Washington, Seattle, WA, USA (Monday, February 8 at 1:12 p.m. EST)
  
  
• Single-dose AAV9-CLN6 gene transfer slows the decline in motor and language function in variant late infantile neuronal ceroid lipofuscinosis 6: Interim results from phase 1/2 trial – Emily de los Reyes, MD, Pediatric Neurology, Nationwide Children’s Hospital, Columbus, OH, USA (Wednesday, February 10 at 11:24 a.m. EST)
  
  
• Utilization of artificial intelligence to identify undiagnosed Fabry disease patients: Development of a validated machine learning model – John Jefferies, MD, MPH, Division of Cardiovascular Disease, University of Tennessee Health Science Center, Memphis, TN, USA (Thursday, February 11 at 1:00 p.m. EST)
  

ePoster Sessions:

Monday, February 8, 2:30-3:30 p.m. EST

• Direct intercellular cross-correction of α-galactosidase-A deficiency in Fabry disease podocytes through tunneling nanotubes in a mixed cell culture model – Behzad Najafian, MD, Department of Pathology, University of Washington, Seattle, WA, USA (Poster #169)
  

Tuesday, February 9, 2:30-3:30 p.m. EST

• Functional analysis and clinical curation of human acid alpha glucosidase (GAA) variants of unknown significance (VUS) screened from infants diagnosed with Pompe disease via newborn screening (NBS) – Shelly Goomber, PhD, Division of Medical Genetics, Duke University Medical Center, Durham, NC, USA (Poster #87)
  
  
• Development of a Fabry disease screening tool for chronic pain patients - step 1: Categorization based on phenotypic risk profiles – Michael A. Ueberall, MD, Pediatric Neurology, Institute of Neurological Sciences, Nurnberg, Germany (Poster #256)

Wednesday, February 10, 2:30-3:30 p.m. EST

• Long-term treatment with migalastat 150 mg every other day is associated with sustained cardiac efficacy and is well tolerated – Ulla Feldt-Rasmussen MD, DMSc, Department of Endocrinology, Rigshospitalet, Copenhagen University Hospital, Copenhagen, Denmark (Poster #70)