Sickle Cell Disease News

September 16, 2021September 16, 2021

New Institute Aims to Leave No Rare Disease Patient Behind

A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community with some of the largest ... Read more

September 16, 2021September 16, 2021

Ferriprox Approved in Brazil to Treat Iron Overload in SCD Patients

Sickle Cell Disease News

Regulators in Brazil have approved Ferriprox (deferiprone), Chiesi Global Rare Diseases’ iron-binding oral treatment, for blood transfusion-induced iron overload in people with sickle cell disease (SCD) or other anemias. Ferriprox’s use ... Read more

September 14, 2021September 14, 2021

10th SCD Therapeutics Conference on Sept. 15 to Focus on Advances

COVID-19 News, Sickle Cell Disease News

The 10th Annual Sickle Cell Disease Therapeutics Conference, to be held virtually Sept. 15, will highlight the most recent medical advances and developing treatment trends in the field of sickle ... Read more

September 10, 2021September 10, 2021

Group Focuses on Rare Disease Clinical Trial Participation

Sickle Cell Disease News

Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare disease trial participants are running ... Read more

September 9, 2021September 9, 2021

FDA Giving Priority Review to Oxbryta in Treating Children, 4-11

Sickle Cell Disease News

The U.S. Food and Drug Administration (FDA) has agreed to consider Global Blood Therapeutics’ (GBT) request to expand the use of Oxbryta (voxelotor) to children as young as 4 with sickle ... Read more

September 7, 2021September 7, 2021

FTX-6058 Shows Promise in Raising Fetal Hemoglobin in Healthy Adults

Sickle Cell Disease News

FTX-6058, an investigational oral treatment being developed by Fulcrum Therapeutics for sickle cell disease (SCD) and beta-thalassemia, was found to be effective at raising the levels of fetal hemoglobin in healthy adults enrolled ... Read more

September 3, 2021September 4, 2021

‘Running for Rare’: NORD a Charity Partner of 2021 NYC Marathon

Sickle Cell Disease News

The National Organization for Rare Disorders, known as NORD, was named an official charity partner of the 2021 TCS New York City Marathon, which will be held Nov. 7 both ... Read more

September 2, 2021September 2, 2021

SCD Clinical Trials Network Welcomes First Research Sites

Sickle Cell Disease News

Several clinical research sites seeking to find new treatments for people with sickle cell disease (SCD) are joining a collaborative network created by the American Society of Hematology Research Collaborative ... Read more

August 31, 2021August 31, 2021

Awareness Month Targets Research, Education – and Blood Donations

Sickle Cell Disease News

Since Sept. 21, 1983, when then-President Ronald Reagan’s proclamation created National Sickle Cell Anemia Awareness Month, advocates, patients, family members, doctors, and others impacted by sickle cell disease (SCD) have ... Read more

August 27, 2021August 27, 2021

‘Not Alone in the Dark’: New Guide for, by Caregivers of Children With Rare Diseases

Sickle Cell Disease News

The National Alliance for Caregiving, in partnership with Global Genes, has issued a free guidebook, available online, that offers resources and support for caregivers of children with rare diseases. “The ... Read more

August 26, 2021August 26, 2021

$1M Grant Will Help SCD Patients in Western US Get COVID-19 Vaccine

COVID-19 News, Sickle Cell Disease News

The Sickle Cell Disease Foundation (SCDF) will use a $1 million grant to educate sickle cell disease (SCD) patients living in the western U.S. about the COVID-19 vaccine and assist ... Read more

August 24, 2021August 24, 2021

Sickle Cell Warriors Convention Open to Patients, Families Aug. 25-29

COVID-19 News, Sickle Cell Disease News

The Sickle Cell Consortium, a U.S.-based nonprofit, is hosting a virtual event in late August that will focus on facilitating conversations among people with sickle cell disease (SCD) and their ... Read more

August 19, 2021August 19, 2021

Register Now for Global Genes’ RARE Patient Advocacy Summit

Sickle Cell Disease News

Registration is now open for Global Genes‘ 2021 RARE Patient Advocacy Summit. This year’s hybrid event will be livestreamed from California Sept. 27-29, and some seats also are available for attending ... Read more

August 17, 2021August 17, 2021

Imara Completes Patient Enrollment for Ardent Trial Testing IMR-687

Sickle Cell Disease News

Imara has completed patient enrollment for its Phase 2b Ardent clinical trial, which is testing the safety and efficacy of IMR-687 for the treatment of sickle cell disease (SCD). “We ... Read more

August 12, 2021August 12, 2021

NORD Rare Disease Summit, Online Oct. 18-19, Open for Registration

COVID-19 News, Sickle Cell Disease News

Registration is now open for the 2021 Rare Diseases and Orphan Products Breakthrough Summit, which will be held virtually Oct. 18–19. The event, also known as the National Organization for ... Read more