P021: Long-term follow-up of cipaglucosidase alfa/miglustat in ambulatory and non-ambulatory patients with Pompe disease: An open-label phase I/II study (ATB200-02)

• Priya Kishnani,
• Benedikt Schoser,
• Drago Bratkovic,
• Paula Clemens,
• Ozlem Goker-Alpan,
• Xue Ming,
• Mark Roberts,
• Matthias Vorgerd,
• Kumaraswamy Sivakumar,
• Ans van der Ploeg,
• Mitchell Goldman,
• Jacquelyn Wright,
• Fred Holdbrook,
• Vipul Jain,
• Sheela Sitaraman Das,
• Yasmine Wasfi,
• Tahseen Mozaffar,
• Barry Byrne

Affiliations

Priya Kishnani

Duke University Medical Center, Durham, NC

Benedikt Schoser

Friedrich-Baur-Institut, Neurologische Klinik, Ludwig-Maximilians-Universität München, Germany

Drago Bratkovic

PARC Research Clinic, Royal Adelaide Hospital, Adelaide, SA, Australia

Paula Clemens

University of Pittsburgh School of Medicine and VA Pittsburgh Healthcare System, Pittsburgh, PA

Ozlem Goker-Alpan

Lysosomal and Rare Disorders Research and Treatment Center, Fairfax, VA

Xue Ming

Department of Neurology, Rutgers New Jersey Medical School, Newark, NJ

Mark Roberts

Salford Royal NHS Foundation Trust, Salford, UK

Matthias Vorgerd

University Hospital Bergmannsheil, Heimer Institute for Muscle Research, Bochum, Germany

Kumaraswamy Sivakumar

Neuromuscular Clinic and Research Center, Phoenix, AZ

Ans van der Ploeg

Erasmus MC University Medical Center, Rotterdam, Netherlands

Mitchell Goldman

Amicus Therapeutics, Inc., Philadelphia, PA

Jacquelyn Wright

Amicus Therapeutics, Inc., Philadelphia, PA

Fred Holdbrook

Amicus Therapeutics, Inc., Philadelphia, PA

Vipul Jain

Amicus Therapeutics, Inc., Philadelphia, PA

Sheela Sitaraman Das

Amicus Therapeutics, Inc., Philadelphia, PA

Yasmine Wasfi

Amicus Therapeutics, Inc., Philadelphia, PA

Tahseen Mozaffar

University of California, Irvine, CA

Barry Byrne

University of Florida, Gainesville, FL