Amicus Therapeutics Announces Presentations and Posters at 14th Annual WORLDSymposium™ 2018
Oral Platform Presentations:
Pompe Disease:
- Updated results from ATB200-02: a first-in-human, open-label, phase 1/2 study of ATB200 co-administered with AT2221 in adults with Pompe disease–
Tahseen Mozaffar , MD, Director, Neuromuscular Program,Neurology School of Medicine at UC Irvine (Thursday, February 8 at1:15 p.m. PT )
Fabry Disease:
- Ten years of migalastat treatment in a patient with Fabry disease: a case report –
Raphael Schiffmann , MD,Baylor Research Institute (Thursday, February 8 at1:45 p.m. PT )
Poster Session:
Fabry Disease:
- Pregnancy outcome after exposure to migalastat: a case study – Gere Sunder-Plassmann, MD,
Department of Medicine III ,Division of Nephrology and Dialysis ,Medical University ofVienna ,Vienna, Austria (Poster #129)
- Cardiac outcomes with long-term migalastat treatment in patients with Fabry disease: results from phase 3 trials –
Dominique P. Germain , MD, PhD,Division of Medical Genetics at theUniversity of Versailles and Assistance Publique - Hôpitaux deParis (Poster #LB-18)
Pompe Disease:
- First-in-human preliminary pharmacokinetic data on a novel recombinant acid α-glucosidase, ATB200, co-administered with the pharmacological chaperone, AT2221, in patients with late-onset Pompe disease –
Franklin Johnson , MS,Amicus Therapeutics, Inc. Cranbury, USA . (Poster #168)
Poster Session:
Fabry Disease:
- Renal outcomes with up to 9 years of migalastat in patients with Fabry disease: results from an open-label extension study –
Kathleen Nicholls , MD,Royal Melbourne Hospital (Poster #270)
- A next-generation Fabry enzyme replacement therapy: a proprietary human α-galactosidase A co-formulated with a pharmacological chaperone, AT1001, shows greater substrate reduction than standard of care in Fabry mice –
Su Xu , PhD,Amicus Therapeutics, Inc. Cranbury, USA (Poster #408)
Pompe Disease:
- The patient and clinician point of view: living with late-onset Pompe disease –
Nita Patel, RN , GCN, CCR,Amicus Therapeutics, Inc. Cranbury, USA (Poster #298) - Updated results from ATB200-02: a first-in-human, open-label, phase 1/2 study of ATB200 co-administered with AT2221 in adults with Pompe disease –
Tahseen Mozaffar , MD, Director, Neuromuscular Program,Neurology School of Medicine at UC Irvine (Poster #LB-38)
The goal of the WORLDSymposia is to provide an interdisciplinary forum to explore and discuss specific areas of interest, research, and clinical applicability related to lysosomal diseases. Each year, WORLDSymposia hosts a scientific meeting presenting the latest information from basic science, translational research, and clinical trials for lysosomal diseases. This symposium is designed to help researchers and clinicians to better manage and understand diagnostic options for patients with lysosomal diseases, identify areas requiring additional basic and clinical research, public policy and regulatory attention, and identify the latest findings in the natural history of lysosomal diseases. For more information please visit www.worldsymposia.org.
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Forward-Looking Statements
This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to preclinical and clinical development of our product candidates, The inclusion of forward-looking statements should not be regarded as a representation by us that any of our plans will be achieved. Any or all of the forward-looking statements in this press release may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. For example, with respect to statements regarding clinical trials, actual results may differ materially from those set forth in this release due to the risks and uncertainties inherent in our business, including, without limitation: the potential that results of clinical or preclinical studies indicate that the product candidates are unsafe or ineffective; the potential that it may be difficult to enroll patients in our clinical trials; the potential that regulatory authorities, including the
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Source: Amicus Therapeutics, Inc.