Updates

April 18, 2019April 18, 2019
Loma Linda Hospital ‘Effecively Cures’ Girl Using Stem Cells Transplanted from Dad, Report Says

Clinicians at the Loma Linda University Children’s Hospital successfully performed that institute’s first stem cell transplant, and report having “effectively cured” a girl with sickle cell disease ... Read more
April 16, 2019April 16, 2019
Low Oxygen Regions in Brains of Sickle Cell Patients are Prone to Small Strokes, Study Shows

Sickle cell disease patients experience silent strokes in the deep white matter of the brain that is prone to low oxygen supply, a ... Read more
April 11, 2019April 11, 2019
Abnormal Red Blood Cells’ Shape Affect Blood Clot Formation, Study Shows

Abnormal sickle-shaped red blood cells characteristic of sickle cell disease (SCD) change the structure and dynamics of blood clots’ formations in veins, a ... Read more
April 9, 2019April 9, 2019
Identifying Outcomes for Sickle Cell Disease Clinical Trials is Aim of coreSCD

The Green Park Collaborative (GPC) is working with the Sickle Cell Disease Association of America (SCDAA) to develop a consensus of desired outcomes to be used ... Read more
April 4, 2019April 4, 2019
Pfizer Executive Outlines Vision for Treating Sickle Cell Disease, Other Rare Diseases

Consumers may know Pfizer as the company that manufactures Viagra, but the New York-based pharma giant is investing millions in developing potential treatments and gene ... Read more

News

Study Determines Low Adherence to Hydroxyurea Treatment Among Children, Adolescents
April 2, 2019April 2, 2019
Children and adolescents with sickle cell anemia (SCA) showed low adherence to treatment with ...
News

Alterations in Blood Vessels of the Lungs Should be Monitored in SCD Patients, Study Says
March 28, 2019March 28, 2019
Alterations in the lungs’ network of blood vessels, caused by sickle cell disease (SCD), ...
News

Problem-Solving Intervention May Help SCD Teens to Transition to Adulthood, Study Shows
March 26, 2019March 26, 2019
Development of problem-solving skills may help adolescents with sickle cell disease prepare for the ...
News

More Curative Bone Marrow Transplants are Successful When Patients Given Double the Radiation Beforehand, Study Says
March 21, 2019March 21, 2019
The success rate of bone marrow transplants for patients with sickle cell disease or ...
News

Oral Morphine is a Viable Treatment Option for SCD Patients Experiencing VOCs, Study Says
March 19, 2019March 19, 2019
Oral morphine treatment is a valid therapy option for patients with sickle cell disease ...
News

Brexit Could Have Real Effects for UK Rare Disease Patients, Experts Warn
March 15, 2019March 14, 2019
Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will ...

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Congo Delegation Visits Howard University to Bolster Collaborations on Sickle Cell Disease
March 14, 2019March 14, 2019
A Republic of Congo delegation of health and higher education representatives recently met with ...
NIH Rare Disease Day Highlights Joint Networks Advancing Array of Research
March 13, 2019March 13, 2019
Rare diseases affect about 30 million Americans — roughly the same number as those ...
Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist
March 12, 2019March 12, 2019
Weight-loss Surgery May Help Reduce VOCs in Morbidly Obese SCD Patients
March 12, 2019March 12, 2019
Women with Sickle Cell Disease May Have Earlier Decline of Ovarian Function, Study Suggests
March 7, 2019March 7, 2019
Differences in Brain Oxygen Supply May Explain Silent Strokes in SCD Patients
March 5, 2019March 5, 2019

First Patient Enrolled in Phase 1/2 Trial Testing CTX001 for Severe Sickle Cell Disease
February 28, 2019February 28, 2019
A Phase 1/2 clinical trial evaluating investigational therapy CTX001 for severe sickle cell disease (SCD) has enrolled ...
WODC 2019 Organizers Expect 1,200 to Attend Rare Disease Conference in April
February 26, 2019February 26, 2019
ACCEL Program Launched to Improve Healthcare Access for Sickle Cell Patients
February 21, 2019February 21, 2019
Vienna to Host RARE2019 Meeting on Rare Diseases
February 21, 2019February 21, 2019

GBT Publishes Data from Phase 1/2 Trial of Voxelotor in SCD Patients
February 21, 2019February 26, 2019
Global Blood Therapeutics (GBT) published the results from its Phase 1/2 clinical trial that tested ...
Spectra Optia Device Save Way to Treat Sickle Cell While Using Less Blood Cell Packs, Study Finds
February 19, 2019February 19, 2019
Patients, Family and Friends to Take Part in Variety of Rare Disease Day Events Worldwide
February 14, 2019February 14, 2019

News

ATS Releases Guidelines for Home Oxygen Therapy in Children with Sickle Cell Disease
February 12, 2019February 19, 2019
The American Thoracic Society (ATS) released a set of new guidelines for home oxygen ...
News

Emmaus Unveils Co-payment Assistance Program for SCD Endari Patients
February 7, 2019February 7, 2019
For eligible sickle cell disease (SCD) patients covered by commercial insurance, biopharmaceutical company Emmaus ...
News

FDA Revising ‘Draft Guidance’ on Developing Treatments for Rare Diseases
January 31, 2019January 31, 2019
The U.S. Food and Drug Administration(FDA) is updating its 2015 draft guidelines for drug ...
News

Kids with Sickle Cell Anemia are More Sedentary than Healthy Peers, Study Suggests
January 31, 2019January 31, 2019
Children and adolescents with sickle cell anemia do less physical activity and have a ...
News

Registration Open for Sickle Cell Foundation of Georgia’s Annual Camp New Hope
January 29, 2019January 29, 2019
The Sickle Cell Foundation of Georgia (SCFG) will host its 42nd Annual Camp New ...
News

Vaso-Occlusive Pain Linked to Menstruation in Some Patients, Study Finds
January 24, 2019January 27, 2019
Acute vaso-occlusive pain is associated with the onset of menstruation, and about 25% of ...
News

Safety and Early Hints of Benefit Seen in Phase 1b Trial of PF-04447943
January 22, 2019January 22, 2019
Sickle cell disease (SCD) patients were safely treated with a phosphodiesterase 9A (PDE9) inhibitor ...
News

Howard University President Launches ‘Run To Cure Sickle Cell’ Campaign
January 18, 2019January 18, 2019
Howard University President Wayne A.I. Frederick has unveiled a campaign to raise awareness and funds for the ...
News

Randomized Trials Needed to Assess Benefits of Salmonella Vaccines in SCD Patients, Study Says
January 15, 2019January 15, 2019
Randomized controlled trials are needed to examine the benefits of vaccinating people with sickle ...

October 25, 2016October 25, 2016

Kids with Sickle Cell Disease at Risk for Lead Poisoning, NYC Study Shows

News

National and local policies have led to large reductions in lead poisoning cases overall in the U.S., but severe cases are still common among children. Recently, researchers at the New York City Department of Health and Mental Hygiene (DOHMH) investigated sources of exposure, risk factors, and ... Read more

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