An article in the Proceedings of the National Academy of Sciences (PNAS) Nexus journal* by Goel et al. describes studies in ...

The high cost of sickle cell gene therapies is pushing private insurers and Medicaid programs to develop new payment models.

For the average person, living at high altitude—where oxygen is sparser than at sea level—can have health benefits ranging ...

An SCD patient in Bahrain received the gene-editing therapy Casgevy, marking the first time the treatment has been given outside the U.S.

Novartis has said it is “disappointed” that the EMA’s Human Medicines Committee has recommended revoking the conditional EU approval for sickle cell disease ... offer the drug to patients.

Other drugs have been launched for SCD in recent ... it remains on sale in the US and other markets. The Sickle Cell Society described the decision to withdraw its European marketing authorisation ...

Lawyers described a lifetime of pain management that ultimately led to the death of 20-month-old Reign Nelson.

Despite promising Phase 3 results, safety concerns, namely liver injury, have impacted Agios stock and could affect Pyrukund's approval and market adoption. Read the full report here.

Filed for Regulatory Approval of Mitapivat (PYRUKYND®) for the Treatment of Adult Patients with Non-Transfusion-Dependent and ...

No doubt 2025 will usher in many more advances in CRISPR therapies, thanks to the FDA’s new Platform Technology Designation ...