Skin Conditions

Sep 22, 2021| Posted in: Advocacy, Autoimmune / Autoinflammatory Disorders, Cancers, Congenital And Genetic Conditions, Diagnosis, Drug Development, Endocrine Disorders, Expert Perspectives, 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

What the NORD Breakthrough Summit Will Look Like in 2021

Mary Dunkle of NORD discusses the upcoming Rare…
Sep 21, 2021| Posted in: Advocacy, Autoimmune / Autoinflammatory Disorders, Cancers, Congenital And Genetic Conditions, Diagnosis, Drug Development, Endocrine Disorders, Expert Perspectives, 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

CheckRare Panel Discussion: Data Silos Are Hindering Rare Disease Research

Great conversation with Drs Paul Howard, Eric March,…
Sep 15, 2021| Posted in: Cancers, Drug Development, Lung Diseases, Skin Conditions, Treatment

FDA Gives Accelerated Approval for Zanubrutinib to Treat Marginal Zone Lymphoma

The U.S. Food and Drug Administration (FDA) has…
Sep 9, 2021| Posted in: Cancers, Skin Conditions, Treatment

What Are the Treatment Options for CTCL?

Sima Rozati, MD, Assistant Professor of Dermatology at…
Sep 3, 2021| Posted in: Autoimmune / Autoinflammatory Disorders, Cancers, Congenital And Genetic Conditions, Drug Development, Endocrine Disorders, Gastrointestinal 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

2021 Orphan Drugs: PDUFA Dates and FDA Approvals

Rare diseases and orphan drugs are at the…
Sep 3, 2021| Posted in: Cancers, Skin Conditions

CTCL Presentation Varies Widely Among Patients, Even Within Subtypes

Sima Rozati, MD, Assistant Professor of Dermatology at…
Aug 30, 2021| Posted in: Cancers, Skin Conditions

How CTCL Affects African American Subpopulations Differently

Sima Rozati, MD, Assistant Professor of Dermatology at…
Aug 16, 2021| Posted in: Congenital And Genetic Conditions, Musculoskeletal Diseases, Skin Conditions

FDA Review of Palovarotene to Treat Fibrodysplasia Ossificans Progressiva (FOP) Halted (Temporarily)

The wait for a new therapy to treat…
Aug 11, 2021| Posted in: Congenital And Genetic Conditions, Hematologic Disorders, Skin Conditions, Treatment

Treatment Options for Systemic Mastocytosis

Daniel DeAngelo, MD, PhD, Chief, Division of Leukemia,…
Jul 28, 2021| Posted in: Autoimmune / Autoinflammatory Disorders, Cancers, Hematologic Disorders, Skin Conditions, Treatment

Avapritinib: Mechanism of Action

Daniel DeAngelo, MD, PhD, Chief, Division of Leukemia,…

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.

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.

CME Program: Managing Individuals With Eosinophilic Granulomatosis with Polyangiitis (EGPA)

Michael E. Wechsler, MD, MMSc provides an overview of Eosinophilic Granulomatosis with Polyangiitis (EGPA) – including how to shorten individuals’ diagnostic journey and the time till they access effective therapy – through these brief courses that are certified for CME credit.

Skin Conditions

Topics