Long Chain Fatty Acid Oxidation Disorder (LCFAOD)

Features

Two-Day Virtual Conference Will Explore Fatty Acid Oxidation Disorders

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

Israel Emerges as Global Powerhouse in Rare Disease Research

TEL AVIV, Israel — On the 6th floor of Schneider Children’s Hospital in the Tel Aviv suburb of Petah Tikva, 30 children with Alagille syndrome and 2 with lysosomal acid lipase deficiency (LAL-D) receive specialized treatment from a trio of pediatric hepatologists. Across town, at Sheba Medical Center’s Edmond and Lily Safra Children’s Hospital, pediatric…
Ultragenyx CMO Camille Bedrosian

Improving the Lives of Patients With LCFAOD

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

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. The virtual ceremony was hosted by John Whyte, MD, MPH, chief medical officer at WebMD, with commentary…

LCFAOD Clinical Insights

LCFAOD Differential Diagnosis

Current Approaches to Treating Long Chain Fatty Acid Oxidation Disorder

Long chain fatty acid oxidation disorder (LCFAOD) is a rare and life-threatening disease that is inherited in an autosomal recessive manner. It impairs the body’s production of energy, which leads to chronic energy insufficiency and injury to the organs that depend on it. Although LCFAOD is screened for in newborns, current management strategies still too…
PAH experimental therapies

Carrier Screening for Autosomal Recessive and X-Linked Conditions

Medicine as it is understood today is an umbrella term that covers the detection, diagnosis, management, and prevention of disease. It is, rightly so, akin to a broad kitchen in which there can never be too many cooks. As physicians debate how best to treat genetic rare diseases, (such as Duchenne muscular dystrophy, spinal muscular…

Rare Care Podcast

Perspectives on Long Chain Fatty Oxidation Disorder With Dr. Jerry Vockley 


Ultragenyx CMO Dr. Camille Bedrosian Discusses the First FDA-Approved Treatment for LCFAOD”