Updates

October 1, 2020October 1, 2020
Medications Activating sGC Protein May Have Promise as SCD Therapy, Study Shows

Medications that activate the protein soluble guanylyl cyclase (sGC) may have promise in the treatment of sickle cell disease (SCD), a new study ... Read more
September 30, 2020September 29, 2020
Saying I’m Fine When I’m Not to Avoid the Hospital

Needing to go to the hospital due to a vaso-occlusive crisis is never ideal or pleasant. As a kid, the idea of going to ... Read more
September 29, 2020September 28, 2020
Efforts Continue Beyond Awareness Month to Expand Newborn Screening Panels in US

It took one year for Dona Krystosek to get a diagnosis for her son, Levi, after he was born. The family received three misdiagnoses ... Read more
September 29, 2020September 29, 2020
Gene Therapy LentiGlobin Granted Priority Medicines Designation for SCD in EU

Bluebird Bio’s investigational gene therapy LentiGlobin has been granted priority medicines (PRIME) designation by the European Medicines Agency (EMA) for the treatment of sickle cell disease (SCD). Given to candidate ... Read more
September 24, 2020September 24, 2020
Gene-editing Therapy CTX001 Granted Priority Medicines Designation for Severe SCD in EU

CTX001, an investigational gene-editing cell therapy, has been granted priority medicines (PRIME) designation by the European Medicines Agency (EMA) for the treatment of ... 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

Medications Activating sGC Protein May Have Promise as SCD Therapy, Study Shows

Saying I’m Fine When I’m Not to Avoid the Hospital

Efforts Continue Beyond Awareness Month to Expand Newborn Screening Panels in US

Gene Therapy LentiGlobin Granted Priority Medicines Designation for SCD in EU

Gene-editing Therapy CTX001 Granted Priority Medicines Designation for Severe SCD in EU

I Started Going Outdoors More, but Soon Changed My Mind

Graphite Bio Raises $45M to Move New Way of Gene Editing Into Sickle Cell Trial

Trying a Different Medication to Address Iron Overload

GreenLight Wins $3.3M Grant to Pursue mRNA-based Gene Therapies

Thinking Ahead by Cultivating Healthy Habits Now

Study Identifies FDA-approved Medications With Potential for Treating SCD

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Activities Underway for Newborn Screening Awareness Month

Genetic Differences Seen to Mirror Sickle Cell Severity in African Study

My 2 Hopes for Sickle Cell Awareness Month

‘Brain-Gut Axis’ May Be What Links Stress and Crises in SCD Patients

My Birthday Wish for Sickle Cell Patients

Grant Expands SCD Patient Registry of COVID-19 Infections to Include Africa

What Chadwick Boseman Taught Me About Perseverance

Same But Different Photo Contest Celebrates People With Rare Diseases

Sickle Cell Awareness Month Events Underway Across US

European Commission Grants Orphan Drug Status to Imara’s IMR-687 for Sickle Cell Disease

Breaking the Habit of Assuming the Worst in Others

EDIT-301, Experimental Cell Therapy for SCD, Earns Rare Pediatric Disease Designation

Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input

Medical Marijuana Reduces Pain and Hospital Visits in SCD Patients, Study Indicates

Sickle Cell Patients Should Have Medical Exemption in the UK

First Sickle Cell Patient Dosed in Phase 2b Trial of Imara’s IMR-687

EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US

Rolling With the Punches

Sickle Cell Oral Treatment Xromi Approved in Scotland for Ages 2–9

Parallel, Pitt Team Up to Study Medical Marijuana for Treating SCD Symptoms

New Streaming Channel Showcases Rare Disease Films

What Not to Say to Someone With Sickle Cell

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