Musculoskeletal Diseases

May 21, 2020| Posted in: Musculoskeletal Diseases

Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a progressive form of…
May 20, 2020| Posted in: Congenital And Genetic Conditions, Metabolic Disorders, Musculoskeletal Diseases

Overview of the Mucopolysaccharidoses

Heather A. Lau, MD, Director, Lysosomal Storage Disease…
May 7, 2020| Posted in: Congenital And Genetic Conditions, Expert Perspectives, Musculoskeletal Diseases

Congenital Myotonic Dystrophy

Michael Snape, PhD, Chief Scientific Officer at AMO…
May 4, 2020| Posted in: Musculoskeletal Diseases, Neurology/Nervous System Diseases

Lambert-Eaton Myasthenic Syndrome

Overview Lambert Eaton myasthenic syndrome is a disorder…
May 3, 2020| Posted in: Congenital And Genetic Conditions, Metabolic Disorders, Musculoskeletal Diseases, Neurology/Nervous System Diseases, Ophthalmology/Eye Diseases, Uncategorized

Sanfilippo Syndrome (Mucopolysaccharidosis III)

Other Names: Mucopolysaccharidosis III; Mucopoly-saccharidosis type 3A; Sanfilippo syndrome…
May 1, 2020| Posted in: Congenital And Genetic Conditions, Musculoskeletal Diseases, Ophthalmology/Eye Diseases, Skin Conditions, Uncategorized

Neonatal Onset Multisystem Inflammatory Disease (NOMID)

Other Names: CINCA syndrome; CINCA; Infantile Onset Multisystem Inflammatory…
Apr 27, 2020| Posted in: Congenital And Genetic Conditions, Musculoskeletal Diseases, Neurology/Nervous System Diseases

Hypokalemic Periodic Paralysis

Hypokalemic periodic paralysis (HOKPP) is characterized by episodes of muscle…
Apr 24, 2020| Posted in: Kidney And Urinary Diseases, Musculoskeletal Diseases

Still’s Disease (Adult Onset)

Other Names: Adult Still's disease; Still's disease adult onset.…

CME Program: Mucopolysaccharidoses (MPSs) Highlights from WORLDSymposium 2020

Barbara K. Burton, MD highlights the latest information about MPSs from WORLDSymposium and provides expert analysis of its clinical relevance for members of the care team in order to help them care for patients with these rare diseases.

Rare Disease Clinical Trials CME Program

Learn about clinical trials and rare diseases through these brief courses that are certified for CME and CNE credit.
  • Heather Lau, MD, MS, NYU School of Medicine, New York, NY
  • Morie A Gertz, MD, Mayo Clinic, Rochester, MN
  • Timothy Craig, DO, Penn State University, Hershey, PA
  • Eric T. Wong, MD, Harvard Medical School, Boston, MA

Musculoskeletal Diseases

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