Six-step framework: New guidance combines supportive care, steroids, immunosuppressants, plasma therapy, complement inhibitors, and transplants for C3G. Targeting root cause: FDA-approved and ...

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The expression of 8 complement pathway genes were associated with at least 1 outcome in the patients with IPF. Higher expression levels of membrane-bound complement regulator genes and lower ...

After teasing a new regulatory process for personalized genetic medicines at the end of last year, the FDA today unveiled draft guidance for an approval pathway that could see custom CRISPR therapies, ...

Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...

Also called C3G, this rare kidney disease often affects children and young adults, but it can happen at any age. It has a major impact on a person’s health and life. C3G has two forms: dense deposit ...

NEW YORK, Nov 12 (Reuters) - The U.S. Food and Drug Administration can approve new personalized treatments for rare and deadly genetic diseases based on data from a handful of patients, two of the ...

Elaine Chen covers biotech, co-writes The Readout newsletter, and co-hosts STAT’s weekly biotech podcast, The Readout Loud. You can reach Elaine on Signal at elaineywchen.70. Compass Pathways, which ...

SAN FRANCISCO -- The investigational complement-targeting agent claseprubart was safe and led to significant improvements in generalized myasthenia gravis (MG), the phase II MaGic trial showed. The ...