Post purge, he could finally finish a sentence. Up next, choosing life

Modulator pills transformed cystic fibrosis care. But the fight for a cure is not over — so long as research remains funded.

A channel opens and a breakthrough lets cystic fibrosis patients breathe

After false starts and incremental gains, scientists crack the problem that made breathing impossible. Will answers come fast ...

Study reveals worrying extent of imprecise gene and gene mutation naming

A systematic review of 52 scientific papers submitted to a world-leading clinical genetics journal from multiple scientists ...
NPR

The FDA creates a quicker path for gene therapies

The Food and Drug Administration Monday unveiled the details of a new policy designed to make it easier and quicker for patients with very rare diseases to get cutting-edge treatments. The new ...
Reuters

US FDA proposes framework to speed rare disease gene therapy approvals

WASHINGTON, Feb 23 (Reuters) - The U.S. Food and Drug Administration proposed on Monday a new framework to speed approvals of personalized treatments for rare and life-threatening genetic diseases, ...
FierceBiotech

UPDATED: FDA illuminates new approval pathway for bespoke gene editing therapies

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teasing a new regulatory process for personalized genetic medicines at the end of last year, the FDA today unveiled draft guidance for an approval pathway that could see custom CRISPR therapies, ...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

FDA unveils rules for bespoke gene therapies, predicting flood of rare disease applications

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 ...
The Lancet

Gene therapy for Parkinson's disease: trials and technical advances

cDepartment of Basic and Clinical Neuroscience, King's College London, London, UK dDepartment of Brain Repair and Rehabilitation, Queen Square Institute of Neurology, University College London, London ...