A once-daily oral medication raised annualized height velocity for children and adolescents with achondroplasia, according to ...

Patients may not know that these treatments are experimental or about the risks they may pose. The latest in AJC's ...

Researchers at The University of Texas MD Anderson Cancer Center were able to eradicate measurable residual disease (MRD) in ...

A single infusion of a CRISPR-based gene therapy eliminated painful crises in 27 of 28 patients with severe sickle cell ...

Completion of enrollment anticipated within 12 months GALILEO-3 is intended to support the registration of FLT201 in ...

Tregzi (clinically known as Orca-T) is an allogeneic cellular immunotherapy consisting of purified regulatory and conventional T cells.

Completion of enrollment anticipated within 12 monthsGALILEO-3 is intended to support the registration of FLT201 in the adult GD1 ...

A UVA Health endocrinologist discusses the evidence behind vibration-based devices and what women should know before trying ...

Casgevy sickle cell gene therapy is now FDA-approved for children as young as two, opening access for 5,500 more U.S. kids.

CRISPR Therapeutics wins FDA pediatric approval for Casgevy, boosting market potential and pipeline upside despite slow uptake. Click here to read more.

The FDA on Wednesday expanded the approval for exagamglogene autotemcel (exa-cel, Casgevy) in severe sickle cell disease with ...

I’m currently in the final stages of recovering from osteonecrosis of the jaw (ONJ). Getting a diagnosis took two months, all ...