Dr. Reham Abdel-Wahab of the Cholangiocarcinoma Foundation says a raft of FDA-approved therapies and ongoing clinical trials will expand treatment options.
NORD and other rare disease organizations around the world will celebrate Rare Disease Day on February 28, 2022, with most events being held virtually.
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.
Melinda Bachini, 52, never intended to be a patient advocate for a disease few people have ever heard of, let alone pronounce. But a 2009 diagnosis of cholangiocarcinoma changed everything for the mother of 6.
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.
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.