In the seven months since Vertex Pharmaceuticals’ triple-combo therapy Trikafta (elexacaftor/tezacaftor/ivacaftor) hit the market, people with cystic…
Articles by Larry Luxner
While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has…
European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested…
Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500…
Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500…
As the number of Americans infected with COVID-19 surpasses one million, it’s no surprise that people with rare diseases —…
First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably…
First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably…
Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy…
When Laura Bonnell, founder and president of the Bonnell Foundation, was 12 years old, she decided to become a…