Musculoskeletal Diseases

Jun 1, 2021| Posted in: Autoimmune / Autoinflammatory Disorders, Musculoskeletal Diseases, Neurology/Nervous System Diseases

Myasthenia Gravis

Myasthenia gravis is a chronic autoimmune neuromuscular disease…
May 26, 2021| Posted in: Autoimmune / Autoinflammatory Disorders, Musculoskeletal Diseases

Antisynthetase Syndrome

Antisynthetase syndrome is a chronic autoimmune disease that…
May 25, 2021| Posted in: Advocacy, Congenital And Genetic Conditions, Musculoskeletal Diseases, Neurology/Nervous System Diseases

Huntington Disease Patients and Clinical Researchers Remain Close

Louise Vetter, President and CEO of the Huntington’s…
May 17, 2021| Posted in: Congenital And Genetic Conditions, Musculoskeletal Diseases, Neurology/Nervous System Diseases

Huntington Disease: What Health Care Practitioners Should Know

Louise Vetter, President and CEO of the Huntington’s…
May 15, 2021| Posted in: Advocacy, Congenital And Genetic Conditions, Musculoskeletal Diseases, Neurology/Nervous System Diseases

May Is Huntington Disease Awareness Month

Initiated by the Huntington Society of Canada, Huntington…
May 13, 2021| Posted in: Congenital And Genetic Conditions, Musculoskeletal Diseases, Neurology/Nervous System Diseases, Treatment

The Advantages of Orally Administered Risdiplam to Treat Spinal Muscular Atrophy

Matthew Klein, MD, Chief Development Officer at PTC…
May 5, 2021| Posted in: Congenital And Genetic Conditions, Drug Development, Musculoskeletal Diseases, Neurology/Nervous System Diseases

Mechanism of Action of Risdiplam

Matthew Klein, MD, Chief Development Officer at PTC…
May 5, 2021| Posted in: Congenital And Genetic Conditions, Drug Development, Musculoskeletal Diseases, Neurology/Nervous System Diseases

Radical Change in the Huntington Disease Research Landscape

Michael Hayden, MBChB, PhD, CEO of Prilenia Therapeutics…
May 4, 2021| Posted in: Congenital And Genetic Conditions, Drug Development, Musculoskeletal Diseases, Neurology/Nervous System Diseases, Treatment

Switching from Prednisone to Deflazacort To Treat Duchenne or Becker Muscular Dystrophy

Sue Apkon, MD, Chief of Pediatric Rehabilitation Medicine,…
Apr 30, 2021| Posted in: Congenital And Genetic Conditions, Drug Development, Musculoskeletal Diseases, Neurology/Nervous System Diseases

PROOF-HD Study Enrolling Huntington Disease Patients

Michael Hayden, MBChB, PhD, CEO of Prilenia Therapeutics…

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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