Musculoskeletal Diseases

Jan 18, 2021| Posted in: Congenital And Genetic Conditions, Drug Development, Musculoskeletal Diseases, Skin Conditions

Ipsen Biopharmaceuticals Commitment to Fibrodysplasia Ossificans Progressiva (FOP)

Stephanie Brown, the SVP of rare diseases at…
Jan 17, 2021| Posted in: Advocacy, Congenital And Genetic Conditions, Musculoskeletal Diseases, Treatment

Treating Infantile-Onset Facioscapulohumeral Muscular Dystrophy

Ally Roets and her son Sam — who…
Jan 15, 2021| Posted in: Congenital And Genetic Conditions, Drug Development, Musculoskeletal Diseases, Skin Conditions

Ipsen Biopharmaceuticals Patient-Focused Approach to Rare Diseases

Stephanie Brown, the SVP of rare diseases at…
Jan 14, 2021| Posted in: Advocacy, Congenital And Genetic Conditions, Diagnosis, Musculoskeletal Diseases

Infantile-Onset Facioscapulohumeral Muscular Dystrophy: Mother and Son Share Their Diagnostic Journey

  Ally Roets and her son Sam —…
Jan 10, 2021| Posted in: Cancers, Diagnosis, Endocrine Disorders, Metabolic Disorders, Musculoskeletal Diseases

Acromegaly and Pituitary Tumors

Lisa Nachtigall, MD, Clinical Director, Neuroendocrine and Pituitary…
Jan 4, 2021| Posted in: Autoimmune / Autoinflammatory Disorders, Cancers, Congenital And Genetic Conditions, Drug Development, Endocrine Disorders, Gastrointestinal Diseases, Heart Diseases, Hematologic Disorders, Kidney And Urinary Diseases, Lung Diseases, Lysosomal Storage Disorders, Metabolic Disorders, Musculoskeletal Diseases, Neurology/Nervous System Diseases, News, Ophthalmology/Eye Diseases, Skin Conditions, Treatment

Orphan Drugs Currently Under Review by the FDA

Every potential Food and Drug Administration (FDA) approval…
Dec 26, 2020| Posted in: Cancers, Congenital And Genetic Conditions, Endocrine Disorders, Musculoskeletal Diseases, Neurology/Nervous System Diseases, Treatment

Acromegaly Guidelines and Treatment Options

Roberto Salvatori, MD, Medical Director, Johns Hopkins Pituitary…
Dec 22, 2020| Posted in: Cancers, Congenital And Genetic Conditions, Endocrine Disorders, Metabolic Disorders, Musculoskeletal Diseases, Treatment

Treatment Options for Acromegaly

Roberto Salvatori, MD, Medical Director, Johns Hopkins Pituitary…
Dec 16, 2020| Posted in: Cancers, Congenital And Genetic Conditions, Endocrine Disorders, Metabolic Disorders, Musculoskeletal Diseases, Neurology/Nervous System Diseases, News, Treatment

New Oral Drug for Patients With Acromegaly

Susan Samson, MD, PhD discusses a new treatment…
Dec 13, 2020| Posted in: Cancers, Congenital And Genetic Conditions, Endocrine Disorders, Musculoskeletal Diseases, Neurology/Nervous System Diseases

When to Suspect Acromegaly

Lisa Nachtigall, MD, Clinical Director, Neuroendocrine and Pituitary…

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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