Metabolic Disorders
Apr 14, 2021| Posted in: Congenital And Genetic Conditions, Lysosomal Storage Disorders, Metabolic Disorders, Musculoskeletal Diseases
Deborah Marsden, MD, Global Medical Expert, Medical Affairs,…
The Diagnostic Journey For MPS VII Patients
Apr 9, 2021| Posted in: Congenital And Genetic Conditions, Diagnosis, Metabolic Disorders, Musculoskeletal Diseases, Ophthalmology/Eye Diseases
Deborah Marsden, MD, Global Medical Affairs Lead at…
What is Sly Syndrome
Apr 7, 2021| Posted in: Cancers, Diagnosis, Endocrine Disorders, Metabolic Disorders
Roberto Salvatori, MD, Medical Director, Johns Hopkins Pituitary…
Diagnosing Acromegaly
Apr 2, 2021| Posted in: Autoimmune / Autoinflammatory Disorders, Congenital And Genetic Conditions, Drug Development, Kidney And Urinary Diseases, Metabolic Disorders, Treatment
Chris Peetz, President and CEO of Mirum Pharmaceuticals,…
Efficacy of Maralixibat to Treat Progressive Familial Intrahepatic Cholestasis (PFIC)
Mar 31, 2021| Posted in: Cancers, Endocrine Disorders, Metabolic Disorders, Treatment
Lawrence S. Kirschner, MD, PhD, Professor of Medicine,…
Acromegaly Treatment Options
Mar 30, 2021| Posted in: Congenital And Genetic Conditions, Gastrointestinal Diseases, Kidney And Urinary Diseases, Metabolic Disorders
Tracy McGregor, MD, Director of Clinical Research at…
What is Primary Hyperoxaluria Type 1?
Mar 26, 2021| Posted in: Congenital And Genetic Conditions, Drug Development, Gastrointestinal Diseases, Heart Diseases, Kidney And Urinary Diseases, Metabolic Disorders, Musculoskeletal Diseases, Ophthalmology/Eye Diseases, Treatment
Chris Peetz, President and CEO of Mirum Pharmaceuticals,…
Investigational Drug Could Be First-Ever Approved Treatment for Alagille Syndrome
Mar 25, 2021| Posted in: Cancers, Endocrine Disorders, Metabolic Disorders, Treatment
Roberto Salvatori, MD, Medical Director, Johns Hopkins Pituitary…
Treatment Options for Acromegaly
Mar 22, 2021| Posted in: Congenital And Genetic Conditions, Diagnosis, Kidney And Urinary Diseases, Lysosomal Storage Disorders, Metabolic Disorders
Dawn Laney, MS, CGC, CCRC, Assistant Professor, and…
“Fabry Disease is Sneaky”: What Physicians Ought to Know
Mar 19, 2021| Posted in: Congenital And Genetic Conditions, Kidney And Urinary Diseases, Metabolic Disorders
Mayah, a college student with primary hyperoxaluria type…
PH1 Patient Recalls Her Many Challenges: No Friends, Urine Pouch, Covid-19
