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President and CEO of SCDAA Regina Hartfield discusses how the association will use the funds from the leadership award.

Regina Hartfield: NORD Recognizes SCDAA for Leadership

Investigators observed a potential risk between Black individuals with baseline signs of dehydration, SCT and related mortality over 30 years.

Sickle Cell Trait-Associated Dehydration Linked to Increased Risk of Death

This episode features a conversation with Dr. Amelle Shillington and Jacob and Ashley Wiley after Shillington diagnosed the extremely rare genetic disease that's afflicted Aislynn Wiley, and only 69 other documented cases worldwide. 

The Rare Disease Report Podcast: TANGO2

Sarcoidosis Screening, Clinical Research Improving with Awareness Efforts

ANCA-associated vasculitis may present like sinusitis, but it typically affects multiple organ systems. 

Kenneth Warrington, MD: Identifying ANCA-Associated Vasculitis

Voxelotor is the first and only FDA-approved medication for children with sickle cell disease. 

New Data Published on Voxelotor for Pediatric Sickle Cell Disease Treatment

Mehdi Mirsaeidi, MD, MPH, Director of the ILD and Sarcoidosis Program at the Jacksonville College of Medicine, joins Lungcast to review the rare disease for Sarcoid Awareness Month.

How Treating, Diagnosing Sarcoidosis Is Still Evolving

Because vasculitis can manifest in various ways, Matthew Koster, MD, talks about identifying Granulomatosis with polyangiitis and other forms of the disease.

Matthew Koster, MD: Identifying GPA and Other Forms of Vasculitis

An overview of the progress in hemophilia treatments and global access to gene therapy. 

A Look at Global Hemophilia Treatment: Replacement, Non-Factor and Gene Therapies

The investigative gene therapy achieved significant phase 3 endpoints, after receiving feedback from the FDA on its efficacy outcomes in 2020.

Valoctocogene Roxaparvovec Nearing Potential Hemophilia A FDA Decision

Sickle Cell

Sickle Cell condition center page, resources on the topics of medical news and expert insight into sickle cell disease can be found. Content includes articles, interviews, videos, podcasts, and breaking news on sickle cell research, treatment, and drug development.

Amyloidosis

The HCPLive amyloidosis page is a resource for medical news and expert insights on the rare disease. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments for amyloid buildup, and more.

Duchenne Muscular Dystrophy

On the HCPLive Duchenne muscular dystrophy page, resources on the topics of medical news and expert insight into DMD can be found. Content includes articles, interviews, videos, podcasts, and breaking news on the rare disease, and more.

Huntington Disease

On the HCPLive Huntington disease page, resources on the topics of medical news and expert insight into HD can be found. Content includes articles, interviews, videos, podcasts, and breaking news on Huntington's chorea, and more.

Lysosomal Storage Disorders

Lysosomal storage disorders page, resources on the topics of medical news and expert insight into the rare diseases can be found. Content includes articles, interviews, videos, podcasts, and breaking news on lysosomal disorder disease research, treatment, and drug development.

The Rare Disease Report page is a resource for medical news and expert insights on rare diseases. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments for sickle cell disease, multiple myeloma, and more.

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