Last year’s ice bucket challenge raised the profile of a rare CNS disease, amyotrophic lateral sclerosis (ALS), a form of motor neuron disease. There is a paucity of treatments for this disease and, like most CNS diseases both common and rare, nothing that alters the exorable progression, which in the case of ALS is likely to prove fatal in three to five years. Susan Mcgoldrick from QCTR provides a review of clinical trial challenges in rare CNS diseases.
https://international-pharma.com/wp-content/uploads/2015/04/09.-A-Review-of-Clinical-Trial.pdf