Metabolic Disorders
Apr 14, 2021| Posted in: Congenital And Genetic Conditions, Lysosomal Storage Disorders, Metabolic Disorders, Musculoskeletal Diseases
The Diagnostic Journey For MPS VII Patients
Deborah Marsden, MD, Global Medical Expert, Medical Affairs,… Apr 9, 2021| Posted in: Congenital And Genetic Conditions, Diagnosis, Metabolic Disorders, Musculoskeletal Diseases, Ophthalmology/Eye Diseases
What is Sly Syndrome
Deborah Marsden, MD, Global Medical Affairs Lead at… Apr 7, 2021| Posted in: Cancers, Diagnosis, Endocrine Disorders, Metabolic Disorders
Diagnosing Acromegaly
Roberto Salvatori, MD, Medical Director, Johns Hopkins Pituitary… Apr 2, 2021| Posted in: Autoimmune / Autoinflammatory Disorders, Congenital And Genetic Conditions, Drug Development, Kidney And Urinary Diseases, Metabolic Disorders, Treatment
Efficacy of Maralixibat to Treat Progressive Familial Intrahepatic Cholestasis (PFIC)
Chris Peetz, President and CEO of Mirum Pharmaceuticals,… Mar 31, 2021| Posted in: Cancers, Endocrine Disorders, Metabolic Disorders, Treatment
Acromegaly Treatment Options
Lawrence S. Kirschner, MD, PhD, Professor of Medicine,… Mar 30, 2021| Posted in: Congenital And Genetic Conditions, Gastrointestinal Diseases, Kidney And Urinary Diseases, Metabolic Disorders
What is Primary Hyperoxaluria Type 1?
Tracy McGregor, MD, Director of Clinical Research at… 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
Investigational Drug Could Be First-Ever Approved Treatment for Alagille Syndrome
Chris Peetz, President and CEO of Mirum Pharmaceuticals,… Mar 25, 2021| Posted in: Cancers, Endocrine Disorders, Metabolic Disorders, Treatment
Treatment Options for Acromegaly
Roberto Salvatori, MD, Medical Director, Johns Hopkins Pituitary… Mar 22, 2021| Posted in: Congenital And Genetic Conditions, Diagnosis, Kidney And Urinary Diseases, Lysosomal Storage Disorders, Metabolic Disorders
“Fabry Disease is Sneaky”: What Physicians Ought to Know
Dawn Laney, MS, CGC, CCRC, Assistant Professor, and… Mar 19, 2021| Posted in: Congenital And Genetic Conditions, Kidney And Urinary Diseases, Metabolic Disorders