Farrell High School students are raising awareness for an important cause--donating blood for sickle cell anemia.

Actor turned rare disease advocate Luke Rosen tells how his daughter, Susannah, lives with a KIF1A-associated neurological ...

Patients with CaV2.1 channelopathies face severe and often debilitating symptoms, such as seizures, migraines, tremors, and ...

In November 2023, the UK became the first country in the world to authorise a Crispr gene-editing therapy.

Gene-editing tools like CRISPR have unlocked new treatments for previously uncurable diseases. Now, researchers at the ...

A developmental screening tool may identify young children with SCD who are at risk for academic difficulties, a study found.

Sickle cell disease is caused by a single genetic mutation that alters the shape of red blood cells. While the condition causes severe pain and health complications, carriers gain resistance to ...

ETHealthworld.com brings latest genetic testing news, views and updates from all top sources for the Indian Health industry.

Memantine, a cheaply available drug used for decades to treat Alzheimer's, may help prevent sickle cell crises, especially in ...

NHS Scotland has approved a one-time CRISPR gene therapy for severe sickle cell disease, offering durable reduction in vaso-occlusive crises for eligible patients.

Medicaid is doing a novel payment system for the new, promising and expensive sickle cell treatment. It may become a model ...

A new gene therapy for people who can have a stem cell transplant but do not have a suitable donor has been approved.