LIPIN1 DEFICIENCY

Clinical trials for LIPIN1 DEFICIENCY explained in plain language.

Never miss a new study

Get alerted when new LIPIN1 DEFICIENCY trials appear

Sign up with your email to follow new studies for LIPIN1 DEFICIENCY, keep track of the ones that matter, and come back to a personal dashboard instead of checking manually.

Our safety recommendation!

By submitting, you agree to our Terms of use

Not yet finished but already full! 1

Old drug offers new hope for kids with devastating muscle disease

Disease control Terminated

This study looked back at the use of hydroxychloroquine in children with a rare, severe genetic disorder called Lipin-1 deficiency. The condition causes painful, life-threatening muscle breakdown episodes. Researchers wanted to see if the drug, given on a compassionate basis, was…

Matched conditions: LIPIN1 DEFICIENCY

Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Disease control

Last updated Apr 02, 2026 06:57 UTC

LIPIN1 DEFICIENCY

Get alerted when new LIPIN1 DEFICIENCY trials appear

Old drug offers new hope for kids with devastating muscle disease