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September 12, 2019September 12, 2019

Walks, Blood Drives Among Activities Marking Sickle Cell Awareness Month

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From walks to blood drives, a host of activities and efforts are marking this year’s National Sickle Cell Awareness Month, observed annually each September. For its part, Nationwide Children’s Hospital, ... Read more

September 10, 2019September 10, 2019

Voxelotor Before FDA for Approval as Possible Disease-modifying Treatment for Sickle Cell

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The U.S. Food and Drug Administration (FDA) is giving priority review, as requested, to Global Blood Therapeutics‘ application to approve voxelotor as a treatment for sickle cell disease (SCD). Voxelotor, formerly known ... Read more

September 5, 2019September 5, 2019

8th Annual SCD Therapeutics Conference Set for Sept. 10

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Taking place during National Sickle Cell Awareness Month, the 8th Annual Sickle Cell Disease (SCD) Therapeutics Conference will focus on the latest medical advances and developing treatment trends. To be ... Read more

September 3, 2019September 3, 2019

Sickle Cell Trait Doesn’t Predict Cognitive Decline in African Americans, Study Finds

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African Americans who inherit one sickle cell gene and one normal gene — a condition known as sickle cell trait — are not at greater risk of cognitive decline ... Read more

September 2, 2019September 2, 2019

‘Disorder’ Film Festival Offers Look at Siblings Touched by Rare Diseases

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Rare diseases deeply affect not only the children who experience them, but also their healthy brothers and sisters, as their parents can attest. Two entries in November’s “Disorder: ... Read more

August 29, 2019August 29, 2019

At NY Genome Center, Legal Expert Presents Ethical Dilemmas in Gene Editing

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Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such ... Read more

August 29, 2019August 29, 2019

New Computer Model May Be Used to Find Medicines for Sickle Cell Disease Patients

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A new computer model is able to simulate how red blood cells become misshapen in patients with sickle cell disease. The model may be useful in identifying which medicines could ... Read more

August 27, 2019August 27, 2019

Nigerian-British Rugby Player Ade Adebisi Named ‘Ambassador’ for BioMedomics’ Diagnostic Tool

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The former professional rugby player Ade Adebisi, a sickle cell disease patient and advocate, has been named brand ambassador for Sickle Scan, BioMedomics’ rapid disease diagnostic tool. Currently vice chairman and general manager ... Read more

August 22, 2019August 22, 2019

ReachBio Launches Screening Platform to Find New Therapies for SCD

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ReachBio Research Labs has created a drug screening platform to discover therapeutic compounds that trigger the production of fetal hemoglobin (HbF) to treat sickle cell disease (SCD) and other ... Read more

August 20, 2019

Nebraska’s Neena Nizar Seeks Cure for Jansen’s, One of World’s Rarest Diseases

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Imagine living your whole life with a painful disease so rare that only 25 others worldwide have what you have. And that you’re one of just six such people ... Read more