Ohio lawyer Eileen Sullivan Baker became an advocate for people with LCFAOD after her son, John, was diagnosed with the disease.
The Alliance for Regenerative Medicine aims to help in the development of safe and effective regenerative therapies that can benefit rare disease patients.
Ultragenyx CEO Dr. Emil D. Kakkis said federal regulators should focus more on biomarkers and less on clinical endpoints when it comes to drug approvals.
Patients with long chain fatty acid oxidation disorder share their stories as experts race to find better treatments for this rare disease.
Eliminating geographic and ethnic bias in clinical trials for rare diseases is the goal of Jeeva Informatics, says founder and CEO Harsha K. Rajasimha, PhD.
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,…
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.
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…
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.
As the super-contagious Delta variant of COVID-19 sweeps across the globe, driving up infection rates as well as deaths, top US health officials are urging everyone 12 and older—including the country’s 30 million or so rare disease patients—to get vaccinated quickly.