FDA approves first-of-its kind targeted RNA-based therapy to treat a rare disease

First treatment for the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adult patients

The U.S. Food and Drug Administration today approved Onpattro (patisiran) infusion for the treatment of peripheral nerve disease (polyneuropathy) caused by hereditary transthyretin-mediated amyloidosis (hATTR) in adult patients. This is the first FDA-approved treatment for patients with polyneuropathy caused by hATTR, a rare, debilitating and often fatal genetic disease characterized by the buildup of abnormal amyloid protein in peripheral nerves, the heart and other organs. It is also the first FDA approval of a new class of drugs called small interfering ribonucleic acid (siRNA) treatment.

https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm616518.htm

FDA approves new treatment for a rare genetic disorder, Fabry disease

The U.S. Food and Drug Administration today approved Galafold (migalastat), the first oral medication for the treatment of adults with Fabry disease. The drug is indicated for adults with Fabry disease who have a genetic mutation determined to be responsive (“amenable”) to treatment with Galafold based on laboratory data. Fabry disease is a rare and serious genetic disease that results from buildup of a type of fat called globotriaosylceramide (GL-3) in blood vessels, the kidneys, the heart, the nerves and other organs.

https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm616598.htm

FDA permits marketing of transcranial magnetic stimulation for treatment of obsessive compulsive disorder

For Immediate Release

August 17, 2018

Today, the U.S. Food and Drug Administration permitted marketing of the Brainsway Deep Transcranial Magnetic Stimulation System for treatment of obsessive compulsive disorder (OCD).

“Transcranial magnetic stimulation has shown its potential to help patients suffering from depression and headaches,” said Carlos Peña, Ph.D., M.S., director of the Division of Neurological and Physical Medicine Devices in the FDA’s Center for Devices and Radiological Health. “With today’s marketing authorization, patients with OCD who have not responded to traditional treatments now have another option.”

https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm617244.htm

>>2670549

I think it is good. As someone slowly dying of a rare disease, I wish there were a treatment.

>>2670598

Thank you calendaranon