rare diseases
| 3 min read
When science, manufacturing, and regulatory alignment move together, breakthroughs reach patients faster than ever before.
| 4 min read
Vaccination guidances, promising trial outcomes, funding bills and more led the news this week.
| 4 min read
LYMPHIR delivers targeted therapy for a rare and incurable form of skin lymphoma while opening doors for broader immuno-oncology applications.
| 4 min read
Radiprodil is in development as the first therapy designed to directly address the underlying biology of GRIN-related neurodevelopmental disorder.
| 3 min read
People with erythropoietic protoporphyria (EPP) must go to great lengths to avoid sun exposure. There have been no approved treatments that address the disease mechanism, but a new drug could change that.
| 4 min read
An FDA rejection, Takeda restructuring, Trump initiatives and more lead the news this week.
| 4 min read
AI is making studies more patient-centric, data-driven, and adaptable, ensuring patients can find, join, and stay in the trials that matter most.
| 2 min read
First DPP1 inhibitor drug milestone marks a long-awaited breakthrough for NCFB patients.
| 4 min read
For rare and inherited kidney diseases, genetic testing is solving the clinical trial recruitment crisis and reshaping how precision therapies get to patients.
| 5 min read
A wave of FDA rejections reveals how CMC issues are becoming a leading barrier to approval in cell and gene therapy development.
