Hematologic Disorders

Jun 22, 2022| Posted in: Advocacy, Autoimmune / Autoinflammatory Disorders, Cancers, Congenital And Genetic Conditions, Diagnosis, 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, Ophthalmology/Eye Diseases, Regulations, Skin Conditions, Treatment

New Report Estimates the Number of Rare Diseases Is More Than 10,000 

Kirk Lamoreaux, discusses ‘The Power of Being Counted,’…
Jun 16, 2022| 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, Ophthalmology/Eye Diseases, Regulations, Skin Conditions, Treatment

NORD’s Rare Disease Centers of Excellence Benefits Individuals and Communities

Dr. Priya Kishnani at Duke University School of…
Jun 13, 2022| Posted in: Cancers, Diagnosis, Drug Development, Hematologic Disorders, Treatment

Positive Updated Results from MajesTEC-1 Study in Relapsed/Refractory Multiple Myeloma

Dr. Ajay  Nooka, of the Emory School of…
Jun 8, 2022| Posted in: Cancers, Hematologic Disorders, Treatment

Latest Results from the CARTITUDE-2 Study Testing Cilta-cel in Multiple Myeloma Patients

As Dr. van de Donk explains, overall response…
May 25, 2022| Posted in: Advocacy, Autoimmune / Autoinflammatory Disorders, Cancers, Congenital And Genetic Conditions, Diagnosis, 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, Ophthalmology/Eye Diseases, Regulations, Skin Conditions, Treatment

11 Rare Disease Research Working Groups In Development 

Dr Edward Neilan of NORD discusses how the…
May 23, 2022| 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, Ophthalmology/Eye Diseases, Regulations, Skin Conditions, Treatment

NORD’s Centers of Excellence Addresses Unmet Medical Needs

As Dr. Bodamer explains, one of the goals…
May 10, 2022| Posted in: Advocacy, Autoimmune / Autoinflammatory Disorders, Cancers, Congenital And Genetic Conditions, Diagnosis, 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, Ophthalmology/Eye Diseases, Regulations, Skin Conditions, Treatment

Rare Diseases Cost 10 Times More Than Common Diseases 

The overall cost estimate for all rare disease…
May 4, 2022| Posted in: Congenital And Genetic Conditions, Diagnosis, Hematologic Disorders, Lysosomal Storage Disorders, Neurology/Nervous System Diseases, Treatment

Learn About Gaucher Disease and Obtain CME Credit

Dr. Gregory Grabowski leads a CME program focused…
Apr 21, 2022| 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, Ophthalmology/Eye Diseases, Skin Conditions, Treatment

Podcast: The NORD Rare Disease Centers of Excellence

Edward Neilan, MD, PhD, at the National Organization…
Apr 14, 2022| Posted in: Drug Development, Hematologic Disorders

Data from Phase 3 Gene Therapy Trial in Hemophilia B Patients Very Encouraging

Steven Pipe, MD, at the University of Michigan,…

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

Topics