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Supported by an educational grant from Inozyme Pharma; Program: Generalized Arterial Calcification of Infancy (GACI) is a rare mineralization disorder
caused by mutations in the ENPP1 or ABCC6 genes, affecting the circulatory system in
addition to other body systems. Those individuals who survive into adulthood often have
side effects of the disease, including Autosomal Recessive Hypophosphatemic Rickets
Type 2 (ARHR2). Monoallelic Heterozygous ENPP1 Deficiency affects the ability to
regulate calcium and phosphate levels leading to decreased inorganic pyrophosphate
(PPi), and to increased calcium deposition in the vessel wall. Another clinical phenotype
of ENPP1 Deficiency includes Ossification of the Posterior Longitudinal Ligament
(OPLL), which may also be associated with hypophosphatemic rickets, and other
skeletal conditions.
In this live in-person CME-accredited satellite symposium, two rare mineralization
disorder experts will review clinical phenotypes of ENPP1 deficiency including GACI,
ARHR2, OPPL and others by providing guidance for treatment initiation, ending with a
discussion of emerging treatment options, to enhance the clinician’s confidence in
managing patients with these disorders. Speakers include: David R. Weber, MD, Children’s Hospital of Philadelphia, United States and Carlos Ferreira, MD, NIH, United States
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Speaker: Thomas Clemens, PhD, University of Maryland School of Medicine, United States
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Speakers: Thomas Ambrosi, PhD, UC Davis, United States; Matthew Greenblatt, MD, PhD, Weill Cornell Medical College, United States; and
Francesca Gori, PhD, Harvard School of Dental Medicine, United States
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Speakers: Biotech - Cheng- Chia (Kathy) Tang, PhD; Pharma - Yves Sabbagh, PhD; Education Research - Alexandra Werth, PhD; Government/NIH - Kristy Nicks, PhD; Clinician-Scientist - Benjamin Levi, MD; Deborah Veis, MD, PhD, Journal Editor
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Speakers: Lorenz Hofbauer, MD, TU Dresden University Medical Center, Germany; Ian Reid, MD, University of Auckland, New Zealand;
Adina Draghici, PhD, Harvard Medical School, United States
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Speakers: Dongsu Park , PhD, Baylor College of Medicine, United States; Tri Phan, FRACP, PhD, The Garvan Institute, Australia
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Speaker: Serge Ferrari, MD, Geneva University Hospital and Faculty of Medicine, Switzerland
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Speakers: US Perspective, Courtney Karner, PhD, University of Texas Southwestern Medical Center, United States; European/Australia Perspective, Michelle McDonald , PhD, The Garvan Institute of Medical Research, Australia
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Speaker: Jane Cauley, PhD, University of Pittsburgh, United States
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Speaker: Felicia Cosman, MD, Columbia University College of Physicians and Surgeons, United States
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Networking Break in Hall
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4 Concurrent Oral Sessions
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Amgen, Inc. Sponsored Program
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Supported by an educational grant from Ipsen Biopharmaceuticals, Inc.; Program - Fibrodysplasia ossificans progressiva (FOP) is a progressive and permanently disabling
disorder of extraskeletal ossification characterized by episodic and painful flare-ups and
irreversible heterotopic ossification in muscles, tendons, and ligaments. Given the
heterogeneity of disease presentation, FOP may be misdiagnosed as cancer or fibrosis,
leading physicians to order biopsies which can exacerbate the growth of the FOP bone.
Malformed toes or thumbs help distinguish this rare disorder from other skeletal
problems. With a prevalence of approximately 1.3 individuals per million lives, increasing
clinician awareness of signs and symptoms for an early diagnosis is key to helping
control clinical manifestations.
In this live CME-accredited presentation, two FOP experts and prominent research
leaders will discuss strategies for improving early detection of FOP by recognizing
clinical signs and symptoms, review the genetics and causes associated with the
disease, and provide the latest clinical trial data describing potential treatments for the
prevention of heterotopic ossification (HO) in patients with FOP. Speakers include: Matthew Drake, MD, PhD, Mayo Clinic, United States and Angela M. Cheung, MD, PhD, FRCPC, University of Toronto, ON, Canada
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Supported by an educational grant from Kyowa Kirin Pharmaceuticals - PROGRAM: X-linked hypophosphatemia (XLH) is the most common cause of inherited phosphate
wasting and is associated with severe complications resulting from unresolved childhood
symptoms, including disease progression. Diagnosis and specific treatment are
frequently delayed leading to poorer patient outcomes.
During this live in-person CME satellite symposium, our two presenters, including the
Chair of the new global guidelines based on Grade methodology, and endorsed by
several global organizations including the ASBMR and the Endocrine Society, will offer a
review of the final guidelines for clinical practice, namely strategies for early diagnosis
and management, including a narrative on who and how to treat, assessing patient
complications and ongoing monitoring, and closing with a review of available and
emerging therapeutic options to improve treatment selection. SPEAKERS INCLUDE Aliya Khan, MD, FRCPC, FACP, FACE, McMaster University, Canada; Jill H. Simmons, MD, Vanderbilt University Medical Center, United States
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Speakers: Jerome Noailly, PhD, Pompeu Fabra University, Spain and Ricardo Dent, MD, Angitia Incorporated Limited, United States
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SPONSORED BY ALEXION, ASTRAZENECA RARE DISEASES: An examination of hypophosphatasia, including how it impacts the body, an approach to diagnosis, and a potential treatment option. SPEAKER: Eric Rush, MD, FAACP, CCD, Children's Mercy Hospital, University of Kansas Health System, United States.
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4 Concurrent Oral Sessions
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Speakers: Erik A. Imel, MD, MS, Indiana University School of Medicine, United States; Salvatore Minisola, MD, University of Rome, Italy; Iris Hartley, MD, NIH, United States
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Speakers: Douglas Kiel, MD, PhD, Harvard Medical School, United States; Christopher Hernandez, PhD, University of California, San Francisco, United States; and Roberto Pacifici, MD, Emory University, United States
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Speakers: Anshu Banerjee, MD, PhD, World Health Organization, Switzerland and Jotheeswaran Amuthavalli Thiyagarajan, PhD, World Health Organization, Switzerland
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