Updates

February 13, 2020February 13, 2020
Pediatric SCD Patients with Acute Kidney Injury Face More Hospital Care, Higher Costs, Large–scale Analysis Finds

Pediatric patients with sickle cell disease complicated by acute kidney injury (AKI) were more likely to be admitted to the intensive care unit ... Read more
February 12, 2020February 10, 2020
Encounters with Medical Students Can Be Upsetting

My hospital also is a university full of medical students. Trainees follow their supervisors from patient to patient to get a sense of how their ... Read more
February 11, 2020February 11, 2020
FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar

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. ... Read more
February 11, 2020February 11, 2020
Hospital Readmissions High in Sickle Cell Patients, Study Shows

More than a quarter of patients with sickle cell disease (SCD) are readmitted to the hospital within 30 days of being discharged, most ... Read more
February 6, 2020February 6, 2020
With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus

The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business ... Read more

News

Most U.S. Residents Support Human Genetics Research, Online Poll Says
February 6, 2020February 6, 2020
A recent survey found that U.S. residents are largely interested in and support continued ...
Columns, Her Sickled Journey – a Column by Tito Oye

Have You Joined the Sickle Cell Disease News Forums Yet?
February 5, 2020February 5, 2020
For most of my life, I hid the fact that I have sickle cell ...
News

SCD Patients Receiving Hydroxyurea in the U.S. Still Face Many Challenges
February 4, 2020February 4, 2020
People with sickle cell disease (SCD) treated with hydroxyurea in the U.S. still face ...
News

FDA-Approved Therapies for Sickle Cell Disease May Not be Cost-Effective, ICER Draft Report Says
January 30, 2020January 30, 2020
FDA-approved therapies for sickle cell disease (SCD) may not be cost-effective at their current ...
Columns, Her Sickled Journey – a Column by Tito Oye

Sickle Cell’s Prognosis Taught Me to Live with Purpose
January 29, 2020January 28, 2020
We were nearing the end of a biology lesson. In an attempt to excite ...
News

Jasper Raises More Than $50M to Advance Its Potentially Safer Conditioning Therapy for Stem Cell Transplant
January 28, 2020January 28, 2020
Jasper Therapeutics has raised an additional $14.1 million to finance the development of its ...

Subscribe to our mailing list

Get regular updates delivered to your email box.

Thanks! Please check your email for a confirmation email.

FDA Names Potential Gene Therapy for Sickle Cell an ‘Orphan Drug,’ Aruvant Announces
January 23, 2020January 23, 2020
A potential gene therapy for sickle cell disease (SCD) and beta-thalassemia, known as ARU-1801, ...
The Importance of Better Representation in the Medical Industry
January 22, 2020January 27, 2020
Sickle cell disease is a condition that mostly affects people from an African or ...
FT-4202, FORMA’s Experimental SCD Therapy, Fares Well in Phase 1 Trial of Healthy Volunteers
January 21, 2020January 21, 2020
Sickle Cell Patient Describes ‘Rebirth’ After Gene Therapy
January 16, 2020January 21, 2020
More Life Hacks for Managing Sickle Cell
January 15, 2020January 14, 2020
Emmaus Partners with US Bioservices to Offer SCD Patients New Treatment Adherence Program
January 14, 2020January 14, 2020

FDA Grants Rare Pediatric Disease Status to Investigational Gene Therapy ARU-1801 for Sickle Cell
January 9, 2020January 9, 2020
The U.S. Food and Drug Administration (FDA) granted rare pediatric disease designation to ARU-1801, ...
This Is What a Hospital Stay Looks Like
January 8, 2020January 6, 2020
GBT and Syros Partner to Develop Sickle Cell Treatments That Boost Fetal Hemoglobin
January 7, 2020January 22, 2020
Top 10 Sickle Cell Disease Stories of 2019
January 2, 2020January 3, 2020

NIH Grant Backs Project to Develop New Pain Therapy for SCD Patients
December 19, 2019December 19, 2019
The National Institutes of Health (NIH) has awarded a grant to researchers at Carnegie Mellon ...
These Apps Help Me Manage Sickle Cell
December 18, 2019December 16, 2019
California Earmarks $15M for Expanding Care of Adult SCD Patients
December 17, 2019December 17, 2019

News

Gene Therapy LentiGlobin Continues to Show Promising Results, Phase 1/2 Trial Data Shows
December 12, 2019December 12, 2019
LentiGlobin, Bluebird Bio’s investigational gene therapy for sickle cell disease (SCD), continues to show ...
News

Gene Therapy Takes Center Stage at 2019 NORD Summit
December 10, 2019December 10, 2019
With so much recent publicity surrounding gene therapy, it’s no surprise that the topic ...
News

Novartis Partners with Ghana to Treat SCD, Offering Hydroxyurea and Opening Adakveo Trial
December 10, 2019December 18, 2019
Ghana is working with Novartis to make hydroxyurea treatment available to people with sickle cell disease, ...
News

Adakveo Being Tested in Series of Trials, Including at Higher Dose and in Children
December 5, 2019December 5, 2019
A Phase 3 trial is testing a higher dose of Adakveo (crizanlizumab), the first ...
Columns, Her Sickled Journey – a Column by Tito Oye

My Little Helpers for Managing Sickle Cell
December 4, 2019December 2, 2019
Living with sickle cell disease presents many challenges. Whether I’m at home or traveling, ...
News

HHS Secretary Alex Azar Touts White House Efforts to Fight Sickle Cell Disease
December 3, 2019December 3, 2019
By 2029, Americans with sickle cell disease (SCD) could be living 10 years longer ...
Columns, Her Sickled Journey – a Column by Tito Oye

Tips to Handle Long Waiting Times in the Emergency Room
November 27, 2019November 25, 2019
When we have a crisis or another sickle cell-related complication, we often require urgent ...
News

Oxbryta to Enter ‘Confirmatory’ Trial in Sickle Cell Children in Coming Weeks, GBT Says
November 27, 2019November 27, 2019
Oxbryta (voxelotor), the first treatment for sickle cell disease that addresses a root cause, will ...
News

FDA Approves Oxbryta, 1st Treatment Targeting Root Cause of Sickle Cell
November 26, 2019November 27, 2019
Oxbryta (voxelotor), a first sickle cell treatment that works to stop the sickling and ...

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