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LCFAOD Features

Jerry Vockley
Features, LCFAOD Features

Two-Day Virtual Conference Will Explore Fatty Acid Oxidation Disorders

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The world’s signature academic event for scientists who study fatty acid oxidation disorders (FAODs) is scheduled to take place virtually October 27-28, 2021. The two-day annual conference is hosted by INFORM, the International Network for Fatty Acid Oxidation Research and Management.  In previous years, 100 to 200 people have attended the meeting, said Jerry Vockley, MD, PhD,…

Hagit Baris-Feldman
AATD Features, ALGS Features, CAD Features, CCA Features, CF Features, DMD Features, Features, GIST Features, HEM Features, IPF Features, LAL-D Features, LCFAOD Features, MG Features, NMOSD Features, PAH Features, Pompe Features, SCD Features, SMA Features

Israel Emerges as Global Powerhouse in Rare Disease Research

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When it comes to rare disorders, Israel—a New Jersey-sized nation of 9.3 million that’s made headlines this year for its highly successful coronavirus vaccination campaign—is clearly an emerging powerhouse. 

Ultragenyx CMO Camille Bedrosian
Features, LCFAOD Features

Improving the Lives of Patients With LCFAOD

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NOVATO, California — Not long ago, most people with long chain fatty acid oxidation disorder (LCFAOD) had few options for treatment other than snacking on low-fat, high-carbohydrate nutrients every 2 to 6 hours to prevent liver damage. But that has changed in the last 12 months. Triheptanoin (Dojolvi®) on June 30, 2020, became the first…

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NORD Recognizes Progress in Fight Against Rare Disease With 2021 Rare Impact Awards

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Under COVID-19’s lingering shadow, the National Organization of Rare Disorders (NORD) has presented its 2021 Rare Impact Awards to 24 pharmaceutical firms, researchers, politicians, and nonprofit groups for their efforts over the past year on behalf of patients with such illnesses.

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