Kidney And Urinary Diseases

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…
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 16, 2022| Posted in: Congenital And Genetic Conditions, Drug Development, Kidney And Urinary Diseases, Lung Diseases, Lysosomal Storage Disorders, Metabolic Disorders, Musculoskeletal Diseases, Neurology/Nervous System Diseases

Positive Data from Regenxbio’s Gene Therapy Trials for MPS I and MPS II

Steve Pakola, MD discusses data from the ongoing…
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…
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…
Mar 29, 2022| Posted in: Congenital And Genetic Conditions, Drug Development, Heart Diseases, Kidney And Urinary Diseases, Treatment

Gene Therapy vs Enzyme Replacement Therapy for Fabry Disease

Dr. Jerry Vockley, of UPMC Children’s Hospital, discusses…
Mar 25, 2022| Posted in: Diagnosis, Drug Development, Heart Diseases, Hematologic Disorders, Kidney And Urinary Diseases

What Is AL Amyloidosis?

Dr. Jonathan Wall, Co-founder & Interim Chief Scientific…
Mar 24, 2022| Posted in: Advocacy, Autoimmune / Autoinflammatory Disorders, Cancers, Congenital And Genetic Conditions, Diagnosis, 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

The NORD Rare Disease Centers of Excellence

Dr Edward Neilan of NORD discusses how the…

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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