January 21, 2021January 21, 2021
FDA Clears Way for Trial of Gene Editing Therapy for Severe SCD

The U.S. Food and Drug Administration (FDA) has cleared the initiation of a Phase 1/2 trial investigating EDIT-301, Editas Medicine’s experimental gene editing cell therapy ... Read more
January 20, 2021January 20, 2021
NORD’s Caregiver Respite Program Continues Through Pandemic

Caring for a loved one with a rare disease, especially during these uncertain times, demands significant time, attention, patience, and dedication. To help meet ... Read more
January 20, 2021January 18, 2021
I Can Still Be Productive During a Sickle Cell Crisis

I’m writing this column during a sickle cell disease crisis. I have not had one this bad in a while, so I’ve been ... Read more
January 19, 2021January 19, 2021
Clinical Trial Cleared for GPH101, First Potentially Curative SCD Therapy

The investigational gene editing therapy GPH101 will be the first potentially curative treatment for sickle cell disease (SCD) to be tested in a ... Read more
January 14, 2021January 14, 2021
BCH-BB694 Gene Therapy Safely Treating Severe SCD Patients in Pilot Trial

BCH-BB694 — an experimental gene therapy targeting the BCL11A gene — safely increased the levels of fetal hemoglobin and prevented disease-associated complications in six ... 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

FDA Clears Way for Trial of Gene Editing Therapy for Severe SCD

NORD’s Caregiver Respite Program Continues Through Pandemic

I Can Still Be Productive During a Sickle Cell Crisis

Clinical Trial Cleared for GPH101, First Potentially Curative SCD Therapy

BCH-BB694 Gene Therapy Safely Treating Severe SCD Patients in Pilot Trial

Oral IMR-687 Seen to Lessen VOCs, Pain Crises in Patients in Phase 2a Trial

NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ Forum

2020 Was Challenging, but I’m Grateful for My Progress

Oral FT-4202 Safe, Found to Improve Red Blood Cell Function in SCD

Top 10 Sickle Cell Disease Stories of 2020

US Army Begins Testing Recruits for Sickle Cell Trait

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Tips for Staying Healthy This Holiday Season

CTX001 Continues to Prevent VOCs in People With Severe Disease, Trial Finding

Emmaus Support Program Gives US Patients Low-cost Access to Endari

Oxbryta May Be SCD Blood Transfusions Option in Pandemic, Case Report Finds

SCD Patients Benefit from Early Rivipansel Treatment for VOCs, New Analyses Show

GBT Launches Early Access Program for Voxelotor in Europe, Other Regions

The Lessons I’ve Learned as a Columnist

NORD Push for Rare Disease Advisory Councils Focus of Dec. 16 Public Meeting

Hemex Scientists Awarded $100K for Noninvasive Diagnostics Tool for SCD, Other Disorders

Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe

We Go Through Life at Our Own Pace

Gates Foundation Funds Studies Into CRISPR/Cas9 Gene Editing for SCD

Switching Treatments Helped to Reduce My Iron Overload

ARU-1801 Gene Therapy Granted Orphan Drug Status in EU

Why I Will Be Getting the COVID-19 Vaccine

Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry

Rifaximin, Oral Antibiotic, Named Orphan Drug for Potential to Ease Pain Crises

FT-4202, Oral SCD Therapy in Clinical Testing, Named Orphan Drug in EU

‘Disabled’ Is Not a Bad Word

NIH Funds New Project to Improve SCD Care for Children in Malawi

What Does It Mean to Be Successful?

NORD Hosting ‘RareLaunch’ Workshops to Help Start Rare Disease Non-profits

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