Advances and obstacles in rare and neurological disease treatments

This cluster highlights rapid progress in therapies for rare and neurological diseases alongside persistent barriers in getting treatments to patients. Posts cover FDA approval of a new rare disease drug, promising gene therapy for a fatal childhood muscle disorder, and endowed research for the rare PXE condition. They also spotlight innovation momentum in Alzheimer’s drug development and analyses of systemic hurdles in rare disease research and drug access. Together, these stories underscore both scientific breakthroughs and the policy and funding gaps that still need attention.