The European Commission has granted orphan drug status to mitapivat, an oral investigational therapy for sickle cell disease.

One Saturday evening, I was sitting on the sofa with my husband watching a film. Suddenly, I felt a sharp, stabbing pain pierce through my chest. “Babe, call an ambulance,” I uttered just in time ...

Nearly all the patients with severe sickle cell disease (SCD) who were treated with renizgamglogene autogedtemcel (reni-cel) remained free of vaso-occlusive events (VOEs) for up to two years, new data ...

Etavopivat, Novo Nordisk’s investigational oral therapy for sickle cell disease (SCD), may reduce the incidence of vaso-occlusive crises (VOCs) and increase hemoglobin levels. That’s according to data ...

Mary Shaniqua is a sickle cell patient (HbSS) living in the United Kingdom. She was diagnosed with sickle cell at 18 months old and uses “Shaniqua’s Sickle Chronicles” to narrate her experiences, to ...

I’ve previously written about the difficulties I’ve sometimes faced when trying to get my prescriptions filled to treat sickle cell disease. In a stroke of luck about a year ago, I found a pharmacy ...

Most people with sickle cell disease (SCD) who received the gene-editing therapy Casgevy (exagamglogene autotemcel) in clinical trials have been free from vaso-occlusive crises (VOCs) over several ...

Sickle cell disease can manifest as both a visible and invisible illness, depending on the individual and their unique complications. For some, the symptoms are apparent, but for others, the disease ...