Takeda has reported positive results from a phase 3 trial of its experimental therapy TAK-755 for ultra-rare disease congenital thrombotic thrombocytopenic purpura (cTTP), setting up regula
Atara Biotherapeutics has become the first biotech to win regulatory approval for a T-cell therapy based on donated cells, rather than relying on cells harvested from patients and processed
Amicus Therapeutics could be just weeks away from getting EU approval for its new Pompe disease therapy Pombiliti after getting a positive opinion on the drug from the EMA's human medicines
Having recently assessed the relationship between drug price and prevalence of non-oncology rare diseases, CRA’s Life Sciences Practice team explores in greater depth the situation for thes
There are approximately 7,000 different types of rare disease and researchers estimate that there are more than 300 million people worldwide living with such a condition, according to the N