Vertex’s CRISPR treatment for sickle cell disease hits unexpected roadblock

Vertex executives warned that Casgevy, its curative treatment for sickle cell disease, would be slow to reach patients. But ...
Yahoo

ASH24: Leukemia drug sequencing, sickle cell questions and a new kind of CAR-T

This story was originally published on BioPharma Dive. To receive daily news and insights, subscribe to our free daily BioPharma Dive newsletter. In the years since the approval of Imbruvica for ...
Monthly Prescribing Reference

Rilzabrutinib Designated Orphan Drug for Sickle Cell Disease

Rilzabrutinib is an investigational, oral reversible Bruton tyrosine kinase inhibitor. The Food and Drug Administration (FDA) has granted Orphan Drug designation to rilzabrutinib for the treatment of ...
WCIV on MSN

Sickle cell patients ask for individualized care, say MUSC treatment is oversimplified

Sickle cell patients and their families are speaking out, saying their pain is being treated with a one-size-fits-all approach that fails to recognize how diffe ...
CNBC

Healthy Returns: Pfizer pulls sickle cell disease drug from markets – here’s why it matters

A version of this article first appeared in CNBC's Healthy Returns newsletter, which brings the latest health-care news straight to your inbox. Subscribe here to receive future editions. Hello and ...

Safe, low-cost Alzheimer's drug demonstrates efficacy against sickle cell anemia

Memantine, a long-approved and cost-effective drug used to treat Alzheimer's disease, could also benefit patients with sickle cell anemia in the future. Initial clinical data from an international ...
The Observer

Ugandan biotech unveils sickle cell cure millions can afford

At the weekend in Kampala, a piece of news landed that could reshape the future of one of the world’s most neglected diseases. The United States Patent and Trademark Office (USPTO) had accepted a ...
Healio

ASH sickle cell disease research priorities address pain, organ dysfunction, drug development

Please provide your email address to receive an email when new articles are posted on . ASH issued sickle cell disease research priorities that address seven key focus areas. Topics include end-organ ...
Afro

LaRuby May and team of attorneys file suit against Pfizer, claim negligence in premature release of sickle cell drug therapy

Sickle cell awareness month, observed annually in September, is a time to raise awareness about sickle cell disease, a genetic blood disorder that affects millions of people worldwide. The Pfizer drug ...
NBC New York

Healthy Returns: Pfizer pulls sickle cell disease drug from markets – here's why it matters

A version of this article first appeared in CNBC's Healthy Returns newsletter, which brings the latest health-care news straight to your inbox. Subscribe here to receive future editions. Hello and ...