Assessment of the therapeutic effect of IGS2.7, a CK1δ protein kinaseinhibitor, in combination with riluzole for the treatment of ALS-associatedTDP-43 proteinopathy
TTBK1 and CK1 inhibitors restore TDP-43 pathology and avoid disease propagation in lymphoblast from Alzheimer’s disease patients
Neurodegenerative disease treatments are evolving rapidly, combining current therapies with innovative approaches. Discover the latest advances, including Molefy’s research, and explore effective strategies to improve patient outcomes.
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.