Updates

July 7, 2020July 7, 2020
Potential Oral DMT Showing Safety and Signs of Efficacy in Phase 1 Trial

FT-4202 — an investigational treatment for sickle cell disease (SCD) — has a favorable safety profile and the expected biological effects, providing preliminary support ... Read more
July 2, 2020July 2, 2020
7th Annual Walk With the Stars & Move-A-Thon Kicks Off July 11 to Fight SCD

While it will be entirely virtual this year, the 7th Annual Walk With the Stars & Move-A-Thon is taking place July 11 to raise ... Read more
July 1, 2020June 29, 2020
Finding My ‘New Normal’ as Lockdown Restrictions Ease

It has started … the period I have been uncertain about. Restrictions are starting to ease. I have been shielding for over three months, ... Read more
June 30, 2020June 30, 2020
GBT to Request Oxbryta’s Approval in Europe for SCD Patients 12 and Older

Global Blood Therapeutics (GBT) plans to submit a marketing authorization application to the European Medicines Agency (EMA) asking that Oxbryta (voxelotor) be approved for ... Read more
June 25, 2020June 25, 2020
Mitapivat Safely Increases Hemoglobin Levels in SCD Patients, Phase 1 Trial Shows

Mitapivat (AG-348) safely increased the amount of hemoglobin and changed the levels of biomarkers of red blood damage and energy production in adults with ... Read more

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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

Updates

Potential Oral DMT Showing Safety and Signs of Efficacy in Phase 1 Trial

7th Annual Walk With the Stars & Move-A-Thon Kicks Off July 11 to Fight SCD

Finding My ‘New Normal’ as Lockdown Restrictions Ease

GBT to Request Oxbryta’s Approval in Europe for SCD Patients 12 and Older

Mitapivat Safely Increases Hemoglobin Levels in SCD Patients, Phase 1 Trial Shows

On World Blood Donor Day, I Thanked Blood Donors Everywhere

First SCD Patient Treated With CTX001 Remains VOCs-free After 9 Months, Trial Results Shows

Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic

Here Are Ways to Help Friends with Sickle Cell Disease

World Sickle Cell Day Celebrates Patients, Raises Awareness

Let’s Celebrate World Sickle Cell Day by Focusing on Our Achievements

Subscribe to our mailing list

Bluebird Bio Presents Data from LentiGlobin Gene Therapy Trial

Listening to Sickle Cell Patients Is Crucial During a Crisis

GBT to Request That Oxbryta Treat Children Starting at Age 4

Sickle Cell Takes a Toll on My Veins

Coalition Will Address Racial Disparities in Rare Disease Communities

Sickle Cell Society Survey Looks to Better Understand Pandemic’s Affect

Don’t Take Good Health for Granted

IMR-687 Safely Increases Fetal Hemoglobin Levels in SCD Patients, Phase 2a Trial Shows

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

Smartphone App May Help Assess Anemia Using Eyelid Pictures

Vitamin D Supplements May Reduce Pain-related Emergency Room Visits in Children with SCD

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

What’s Inside My Medicine Cabinet?

Partnership to Ensure Supply of LentiGlobin, Potential Gene Therapy

How My Parents Set Me Up for Success in Sickle Cell Management

Sickle Cell Disease Association, Hemanext Join to Support Programs

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

Agios to Decide Next Steps for Mitapivat as SCD Treatment by Mid-year

Rare Diseases Clinical Research Network Opens Online Survey on COVID-19

Gene-editing Therapy CTX001 Granted RMAT Status by FDA for Severe SCD

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

Potential Oral DMT Showing Safety and Signs of Efficacy in Phase 1 Trial

7th Annual Walk With the Stars & Move-A-Thon Kicks Off July 11 to Fight SCD

Finding My ‘New Normal’ as Lockdown Restrictions Ease

GBT to Request Oxbryta’s Approval in Europe for SCD Patients 12 and Older

Mitapivat Safely Increases Hemoglobin Levels in SCD Patients, Phase 1 Trial Shows

On World Blood Donor Day, I Thanked Blood Donors Everywhere

First SCD Patient Treated With CTX001 Remains VOCs-free After 9 Months, Trial Results Shows

Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic

Here Are Ways to Help Friends with Sickle Cell Disease

World Sickle Cell Day Celebrates Patients, Raises Awareness

Let’s Celebrate World Sickle Cell Day by Focusing on Our Achievements

Subscribe to our mailing list

Bluebird Bio Presents Data from LentiGlobin Gene Therapy Trial

Listening to Sickle Cell Patients Is Crucial During a Crisis

GBT to Request That Oxbryta Treat Children Starting at Age 4

Sickle Cell Takes a Toll on My Veins

Coalition Will Address Racial Disparities in Rare Disease Communities

Sickle Cell Society Survey Looks to Better Understand Pandemic’s Affect

Don’t Take Good Health for Granted

IMR-687 Safely Increases Fetal Hemoglobin Levels in SCD Patients, Phase 2a Trial Shows

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

Smartphone App May Help Assess Anemia Using Eyelid Pictures

Vitamin D Supplements May Reduce Pain-related Emergency Room Visits in Children with SCD

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

Video Games Connect Chronically Ill Children Isolated at Home, Hospital

What’s Inside My Medicine Cabinet?

Partnership to Ensure Supply of LentiGlobin, Potential Gene Therapy

How My Parents Set Me Up for Success in Sickle Cell Management

Sickle Cell Disease Association, Hemanext Join to Support Programs

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

Agios to Decide Next Steps for Mitapivat as SCD Treatment by Mid-year

Rare Diseases Clinical Research Network Opens Online Survey on COVID-19

What Does a Sickle Cell Crisis Feel Like?

Gene-editing Therapy CTX001 Granted RMAT Status by FDA for Severe SCD