US Clinics Build Data Hub for Historically Marginalized Sickle Cell Disease
The American Society of Hematology (ASH) is establishing a nationwide learning community to improve the care and quality of life of people with SCD.
SCD Features
Italian Hematologist Urges End to ‘Useless’ Restrictive Diets After Stem Cell Transplants
Hematologist Federico Stella, MD, says restrictive diets for patients in the hospital recovering from stem cell transplants are unnecessary.
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Omidubicel Shows Promise as Replacement for Umbilical Cord Blood in Stem Cell Transplants
Gamida Cell is gearing up to begin sales of omidubicel from Israel to US hospitals for use in stem cell transplants, pending FDA approval of the therapy.
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At 40, Orphan Drug Act Enjoys Rare Bipartisan Support
Rare disease advocates are marking the 40th anniversary of the landmark Orphan Drug Act, which has led to over 1100 approvals for new therapies.
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The Alliance for Regenerative Medicine Fights for Better Cell, Gene Therapies
The Alliance for Regenerative Medicine aims to help in the development of safe and effective regenerative therapies that can benefit rare disease patients.
L-Glutamine in Sickle Cell Disease: The Real-World Impact to Date
L-glutamine was approved in 2017 to treat acute complications in patients with sickle cell disease, but uptake of and adherence to it have been limited.
An Often Overlooked Disease Exiting the Shadows
Funding for sickle cell disease clinical trials and drug development has steadily increased and groundbreaking gene therapy research is being conducted.
Using Technology to Boost Ethnic, Geographic Diversity in Clinical Trials
Eliminating geographic and ethnic bias in clinical trials for rare diseases is the goal of Jeeva Informatics, says founder and CEO Harsha K. Rajasimha, PhD.
CORD Aims to Improve Diagnosis, Drug Access for Canada’s Rare Disease Patients
The Canadian Organization for Rare Disorders advocates for better diagnosis and drug access for Canada’s 3 million people with rare diseases.
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Israel Emerges as Global Powerhouse in Rare Disease Research
When it comes to rare disorders, Israel—a New Jersey-sized nation of 9.3 million that’s made headlines this year for its highly successful coronavirus vaccination campaign—is clearly an emerging powerhouse.
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