Rare disease advocates are marking the 40th anniversary of the landmark Orphan Drug Act, which has led to over 1100 approvals for new therapies.
Hundreds of veterans who served in Vietnam in the 1960s and 1970s are now developing cholangiocarcinoma, likely due to exposure to liver flukes and dioxin.
Durvalumab (Imfinzi®) offers only a modest improvement in long-term survival rates for patients with cholangiocarcinoma, but it is big news.
When 2 friends set out along Spain’s 500-mile Camino de Santiago, they didn’t know how many people they’d inspire with 3 little words: “I’ll Push You.”
A confusing system of disease codes has led to inaccurate reporting of the number of cholangiocarcinoma cases worldwide, a renowned liver specialist said.
Dr. Reham Abdel-Wahab of the Cholangiocarcinoma Foundation says a raft of FDA-approved therapies and ongoing clinical trials will expand treatment options.
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.
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