Hematologic Disorders

Sep 24, 2021| Posted in: Hematologic Disorders, News, Regulations, Treatment

FDA Approves Ruxolitinib to Treat Chronic GVHD

The FDA has approved ruxolitinib (Jakafi)  to treat…
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 22, 2021| Posted in: Cancers, Hematologic Disorders, Treatment

Factors That Impact Treatment Decisions in Elderly Individuals With AML

Dr. Melissa Loh discusses the many factors that…
Sep 21, 2021| Posted in: Cancers, Drug Development, Hematologic Disorders

Will Cilta-Cell Be the Next CAR T-Cell Approval in Multiple Myeloma?

Saad Usmani, MD at Levine Cancer Institute discusses…
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 16, 2021| Posted in: Cancers, Drug Development, Hematologic Disorders

Natural Killer (NK) Cells as a Potential Treatment Option for Multiple Myeloma 

Nina Shah, MD, of the University of California,…
Sep 10, 2021| Posted in: Cancers, Drug Development, Hematologic Disorders, Treatment

18-Month Follow-Up of CARTITUDE-1 Demonstrates Long-Term Efficacy and Safety of Cilta-Cel

Saad Usmani, MD, FACP, Division Chief of Plasma…
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…
Aug 30, 2021| Posted in: Cancers, Congenital And Genetic Conditions, Diagnosis, Endocrine Disorders, Hematologic Disorders, Kidney And Urinary Diseases, Neurology/Nervous System Diseases, Ophthalmology/Eye Diseases, Treatment

FDA Approves Belzutifan to Treat Tumors Associated with von Hippel-Lindau Disease

The Food and Drug Administration (FDA) has approved…
Aug 23, 2021| Posted in: Cancers, Hematologic Disorders

Overview of Sarcomas

Roman Grosiberg, MD, Medical Oncologist and Director of…

CME Program: Gaucher Disease Highlights from WORLDSymposium 2020

Neal Weinreb, MD, FACP highlights the latest information about Gaucher disease from WORLDSymposium and provides expert analysis of its clinical relevance to help health care providers better care for patients with this rare disease.

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.

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

Hematologic Disorders

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