What does obtaining orphan drug designation in Europe mean and why is it key to innovation in ALS?
Orphan drug designation in Europe is a regulatory recognition granted by the EMA to treatments intended for rare diseases, such as ALS. This status provides key incentives—such as market exclusivity and scientific support—that reduce investment risk and accelerate therapeutic innovation, driving the development of new options for patients with urgent unmet medical needs.
The future of ALS research: why the coming years will be decisive
Amyotrophic lateral sclerosis (ALS) is one of the most complex and devastating neurodegenerative diseases of our time. For decades, biomedical research has progressed slowly, with limited therapeutic options and modest clinical impact. However, the landscape is changing. The coming years are shaping up to be a critical turning point for pharmaceutical research in ALS, driven […]