Updates

October 22, 2020October 22, 2020
Siklos, Hydroxyurea-based Treatment for Pediatric SCD, Available in 100 mg Scored Tablets

Siklos (hydroxyurea) — indicated for the treatment of children 2 and older with sickle cell disease — is now available in 100 mg ... Read more
October 21, 2020October 19, 2020
While My Health Challenges Are Tough, I Smile Through Life

One of my favorite features is my smile, and I find that in life, I have many things to smile about. I have very ... Read more
October 21, 2020October 21, 2020
Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its ... Read more
October 20, 2020October 20, 2020
Cyclerion Suspends Development of Olinciguat for Sickle Cell Disease

Cyclerion Therapeutics has decided to suspend the development of olinciguat, its investigational oral therapy for sickle cell disease (SCD), after the treatment failed ... Read more
October 15, 2020October 15, 2020
$7.1M NIH Grant Supports Study of Opioid Alternatives to Manage SCD Pain

The National Institutes of Health (NIH) has awarded a $7.1 million grant to University of Illinois Chicago (UIC) researchers to evaluate the effectiveness of two therapeutic strategies as ... 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

Siklos, Hydroxyurea-based Treatment for Pediatric SCD, Available in 100 mg Scored Tablets

While My Health Challenges Are Tough, I Smile Through Life

Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say

Cyclerion Suspends Development of Olinciguat for Sickle Cell Disease

$7.1M NIH Grant Supports Study of Opioid Alternatives to Manage SCD Pain

C-Path Launches Consortium to Support Sickle Cell Therapy Development

Here’s What Triggers a Sickle Cell Crisis for Me

Rivipansel Granted FDA’s Rare Pediatric Disease Designation for SCD

How to Be a Good Friend to Someone With Sickle Cell

Same But Different Asks Public to Vote on ‘Glimmer of Hope’ Calendar Photos

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Fulcrum’s FTX-6058 Shows Promising Efficacy as SCD Therapy in Disease Models

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

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

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

Siklos, Hydroxyurea-based Treatment for Pediatric SCD, Available in 100 mg Scored Tablets

While My Health Challenges Are Tough, I Smile Through Life

Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say

Cyclerion Suspends Development of Olinciguat for Sickle Cell Disease

$7.1M NIH Grant Supports Study of Opioid Alternatives to Manage SCD Pain

Why I Share My Health Journey on Social Media

C-Path Launches Consortium to Support Sickle Cell Therapy Development

Here’s What Triggers a Sickle Cell Crisis for Me

Rivipansel Granted FDA’s Rare Pediatric Disease Designation for SCD

How to Be a Good Friend to Someone With Sickle Cell

Same But Different Asks Public to Vote on ‘Glimmer of Hope’ Calendar Photos

Subscribe to our mailing list

Fulcrum’s FTX-6058 Shows Promising Efficacy as SCD Therapy in Disease Models

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

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