Rare disease advocates are marking the 40th anniversary of the landmark Orphan Drug Act, which has led to over 1100 approvals for new therapies.
Abstract artist Betty Usdan-Zwickler, 83, of Boca Raton, Florida, is also a fierce advocate for fellow patients with cold agglutinin disease (CAD).
The all-volunteer CAD Foundation fights for patients with a rare anemia that has no approved therapy. Three companies are conducting clinical trials.
Given the rarity of CAD and lack of comparative trials, treatment plans are primarily based on findings from nonrandomized trials and clinical experience.
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.