News

Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review articles covering rare disorders will be available free-of-charge from Elsevier. The offer runs through April 30, and aims to supports work by researchers and clinicians into a better understanding of and treatments for rare diseases, as well…

Starting a 501(c)(3) tax-exempt nonprofit isn’t easy, but the National Organization for Rare Disorders gave a few tips for those  looking to begin the complex process in its Feb. 20 webinar.  William Whitman…

FT-4202, a potential disease-modifying oral treatment for sickle cell disease being developed by Forma Therapeutics, has been given fast track and rare pediatric disease designations by the U.S. Food and Drug Administration (FDA). A fast track status is meant to accelerate the development and review…

The Institute for Gene Therapies (IGT) has launched, with the aim of maximizing the potential of gene therapies in genetic disorders such as sickle cell disease. Comprised of industry leaders, scientists, and patient advocates, the IGT’s overarching goal is to set the foundation for a modernized regulatory…

Acute chest syndrome, a serious complication of sickle cell disease that can accompany a vaso-occlusive crisis, is more likely in SCD patients who run high fevers, and have low blood oxygen levels and poor spleen function during such crises, a study of hospital records suggests. Other possible predictors of…

An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…

African-Americans with sickle cell trait and sickle cell disease have a significantly faster decline in kidney function than their unaffected peers with normal hemoglobin, a study suggests. Men, people with diabetes, and those with good kidney function to start saw kidney function decline fastest, according to…

The Howard University College of Medicine has been awarded a $42,000 grant to support its “LIFE (Learning Is Fundamental for Everyone) with Sickle Cell Disease” project for youth with the inherited blood disorder and their families. The grant, from the Walter Brownley Trust, was awarded to the College of Medicine’s…

Pediatric patients with sickle cell disease complicated by acute kidney injury (AKI) were more likely to be admitted to the intensive care unit (ICU), had longer hospital stays, accumulated greater hospitalization costs, and had a higher rate of readmission, a large-scale analysis…

The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. Yet even with its enormous resources, the FDA these days relies more and more on patients to…