Kidney And Urinary Diseases

Jan 8, 2021| Posted in: Congenital And Genetic Conditions, Heart Diseases, Hematologic Disorders, Kidney And Urinary Diseases, Lysosomal Storage Disorders, Metabolic Disorders, Neurology/Nervous System Diseases, Ophthalmology/Eye Diseases, Skin Conditions

Overview of Fabry Disease

Robert Hopkin, MD, Associate Professor of Clinical Pediatrics…
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…
Nov 23, 2020| Posted in: Congenital And Genetic Conditions, Kidney And Urinary Diseases, Metabolic Disorders

FDA Approves Lumasiran to Treat Primary Hyperoxaluria Type 1

Pritesh J. Gandhi, PharmD of Alnylam Pharmaceuticals provides an…
Nov 23, 2020| Posted in: Congenital And Genetic Conditions, Kidney And Urinary Diseases, Metabolic Disorders

Alnylam, and the PH1 Community, Waiting for the FDA to Approve Lumasiran

Pritesh J. Gandhi, PharmD of Alnylam Pharmaceuticals discusses the…
Nov 10, 2020| Posted in: Autoimmune / Autoinflammatory Disorders, Drug Development, Kidney And Urinary Diseases

Clinical Trials for Lupus Nephritis

Richard A. Furie, MD, from Northwell Health explains…
Nov 2, 2020| Posted in: Cancers, Congenital And Genetic Conditions, Endocrine Disorders, Kidney And Urinary Diseases, Neurology/Nervous System Diseases, Ophthalmology/Eye Diseases, Treatment

MK-6482 to Treat Renal Cell Carcinoma in Von Hippel-Lindau Disease Patients

Eric Jonasch, MD, Professor, Department of Genitourinary Medical…
Oct 27, 2020| Posted in: Cancers, Congenital And Genetic Conditions, Endocrine Disorders, Hematologic Disorders, Kidney And Urinary Diseases, Neurology/Nervous System Diseases, Ophthalmology/Eye Diseases

What is Von Hippel-Lindau Disease?

Eric Jonasch, MD, Professor, Department of Genitourinary Medical…
Oct 15, 2020| Posted in: Congenital And Genetic Conditions, Endocrine Disorders, Expert Perspectives, Kidney And Urinary Diseases, Musculoskeletal Diseases, Treatment

Approval of Burosumab-twza for Treating Tumor-Induced Osteomalacia

Erik Harris, Executive Vice President and Chief Commercial…
Oct 2, 2020| Posted in: Advocacy, Autoimmune / Autoinflammatory Disorders, Cancers, Congenital And Genetic Conditions, Diagnosis, Drug Development, Endocrine Disorders, Featured, 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, Regulations, Skin Conditions, Treatment

A Preview of NORD’s Rare Summit 2020

Peter Saltonstall, President and Chief Executive Officer of…
Sep 4, 2020| Posted in: Congenital And Genetic Conditions, Drug Development, Kidney And Urinary Diseases, Metabolic Disorders, Treatment

Interim Analysis Shows Promise for Nedosiran to Treat Primary Hyperoxaluria

Ralf Rosskamp, M.D., Chief Medical Director at Dicerna…

TTR Amyloidosis CME Program

Morie A Gertz, MD, MACP provides an overview of TTR Amyloidosis – including how to shorten individuals’ diagnostic journey and the time till they access treatment which might improve their quality of life or even prevent disease progression – through these brief courses that are certified for CME credit.

CME Program: Fabry Disease Highlights from WORLDSymposium 2020

Ozlem Goker-Alpan, MD highlights the latest Fabry disease information from the WORLDSymposium and provides expert analysis of its clinical relevance for members of the care team in order to help them care for patients with this rare disease.

WHIM Syndrome Learning Center

WHIM syndrome is a rare, congenital primary immunodeficiency disorder associated with severe neutropenia that affects all ages. However, due to the heterogeneous presentation of the disease, coupled with lack of awareness of the condition, the diagnosis may be delayed, sometimes even into adulthood.

Kidney And Urinary Diseases

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