Updates

September 12, 2019September 12, 2019
Walks, Blood Drives Among Activities Marking Sickle Cell Awareness Month

From walks to blood drives, a host of activities and efforts are marking this year’s National Sickle Cell Awareness Month, observed annually each September. For ... Read more
September 10, 2019September 9, 2019
Sickle Cell 101: Addressing Common Questions About My Blood Disorder

Ed. note: Welcome to the new column, “Her Sickled Journey,” by Tito Oye. Living with sickle cell anemia means that you have unknowingly ... Read more
September 10, 2019September 10, 2019
Voxelotor Before FDA for Approval as Possible Disease-modifying Treatment for Sickle Cell

The U.S. Food and Drug Administration (FDA) is giving priority review, as requested, to Global Blood Therapeutics‘ application to approve voxelotor as a treatment for sickle ... Read more
September 5, 2019September 5, 2019
8th Annual SCD Therapeutics Conference Set for Sept. 10

Taking place during National Sickle Cell Awareness Month, the 8th Annual Sickle Cell Disease (SCD) Therapeutics Conference will focus on the latest medical ... Read more
September 3, 2019September 3, 2019
Sickle Cell Trait Doesn’t Predict Cognitive Decline in African Americans, Study Finds

African Americans who inherit one sickle cell gene and one normal gene — a condition known as sickle cell trait — are not at ... Read more

News

‘Disorder’ Film Festival Offers Look at Siblings Touched by Rare Diseases
September 2, 2019September 2, 2019
Rare diseases deeply affect not only the children who experience them, but also their ...
News

At NY Genome Center, Legal Expert Presents Ethical Dilemmas in Gene Editing
August 29, 2019August 29, 2019
Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is ...
News

New Computer Model May Be Used to Find Medicines for Sickle Cell Disease Patients
August 29, 2019August 29, 2019
A new computer model is able to simulate how red blood cells become misshapen ...
News

Nigerian-British Rugby Player Ade Adebisi Named ‘Ambassador’ for BioMedomics’ Diagnostic Tool
August 27, 2019August 27, 2019
The former professional rugby player Ade Adebisi, a sickle cell disease patient and advocate, has been named brand ...
News

ReachBio Launches Screening Platform to Find New Therapies for SCD
August 22, 2019August 22, 2019
ReachBio Research Labs has created a drug screening platform to discover therapeutic compounds that ...
News

Nebraska’s Neena Nizar Seeks Cure for Jansen’s, One of World’s Rarest Diseases
August 20, 2019
Imagine living your whole life with a painful disease so rare that only 25 ...

Subscribe to our mailing list

Get regular updates delivered to your email box.

Thanks! Please check your email for a confirmation email.

ICER Will Compare Lead Candidate Therapies Crizanlizumab and Voxelotor for Efficacy, Value
August 20, 2019August 22, 2019
An upcoming analysis by the Institute for Clinical and Economic Review (ICER) will compare ...
New Technology May Allow for Better Monitoring of Sickle Cell Disease
August 15, 2019August 15, 2019
A new way to monitor blood cells that doesn’t require microscopic imaging or biochemical ...
Cognitive Decline Not Evident in Black Americans with Sickle Cell Trait, Study Finds
August 13, 2019August 13, 2019
Survival After Kidney Transplant Lower in SCD Patients Than Other Kidney Disease Patients, Analysis Shows
August 8, 2019August 8, 2019
Phase 3 Trial of Rivipansel in Treating SCD Pain Crisis Fails to Meet Goals, Pfizer Announces
August 6, 2019August 6, 2019
Rare Disease Groups Seek Public Support to Renew Newborn Screening Act in Senate
August 1, 2019

Oklahoma Ranks Lowest on Programs Key to Rare Diseases on NORD’s 2019 State Report Card
August 1, 2019
Oklahoma suffers more tornadoes than any other state, has the highest per-capita rate of ...
NHLBI Stepping Up Efforts to Apprise SCD Patients of Therapies and Trials
August 1, 2019August 1, 2019
Emmaus Takes ‘Sideline Sickle Cell’ Campaign to Annual SCD Family and Educational Event
July 30, 2019July 30, 2019
T-cells Promote Inflammation in Children with Sickle Cell as well as Adults, Study Finds
July 25, 2019July 25, 2019

$19.2M Grant to Support SCD Trial into Red Cell Exchange in US and Europe
July 23, 2019July 23, 2019
To lessen the need for hospitalizations and slow or reverse the organ damage seen ...
European Initiative Targets Diagnosis, Treatment of Rare Diseases
July 18, 2019July 18, 2019
FDA Grants Priority Review to Novartis’ Application for Crizanlizumab
July 18, 2019July 18, 2019

News

‘Sickle Cell Speaks’ Campaign Raises Awareness with Aim of Eroding Stigmas, GBT and Partners Say
July 16, 2019July 16, 2019
In partnership with community organizations, Global Blood Therapeutics (GBT) is directing a national campaign ...
News

Non-Invasive Prenatal Screening Test for Sickle Cell Appears Possible, Study Reports
July 11, 2019July 11, 2019
A non-invasive prenatal test to diagnose a risk of sickle cell disease in a baby ...
News

45th Annual SCDAI 8K Walk, Jog, or Bike-a-Thon Set for July 20 in Chicago
July 9, 2019July 9, 2019
The Sickle Cell Disease Association of Illinois (SCDAI) announced plans to hold its 45th ...
News

World’s First Alport Stamp Is Macedonian Mom’s Latest Win for Rare Disease Patients
July 4, 2019
It wasn’t until Gordana Loleska’s son David was 14 years old that doctors in ...
News

Chronic Pain in Sickle Cell Disease Underpinned by Changes in Brain Networks, Study Suggests
July 2, 2019July 2, 2019
Chronic pain in adults with sickle cell disease (SCD), a frequent symptom of the disease, ...
News

Early Treatment with Hydroxyurea Lessened SCD Severity Among Infants, Study Shows
June 27, 2019June 27, 2019
Treatment with hydroxyurea since early infancy, starting at the age of one, is effective ...
News

NORD Honors Industry, Patient Advocates at Rare Impact Awards Gala
June 26, 2019
A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy ...
News

Investigational Oral Inhibitor IMR-687 Shows Promising Results in Ongoing Phase 2 Clinical Trial
June 25, 2019June 25, 2019
Imara’s investigational oral inhibitor IMR-687 is well-tolerated, and shows signs of potential for lowering ...
News

Eurordis Unveils Integrated-care Initiative for Rare Disease Patients
June 20, 2019
Europe’s umbrella organization for 800 rare disease associations has developed a sweeping initiative to ...

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