To bring about a dramatic and wide reaching change in conditions and attitudes for the rare disease community.

https://rarerevolutionmagazine.com/ RARE Revolution Magazine Spring 2026 - Issue 035 Driving change for rare lysosomal storage disorders Bringing you our dedicated issue on all things, lysosomal storage disorders (LSDs). Exploring a group of over 80 rare genetic conditions that impact families worldwide, this edition takes you from the origins of medical discovery to today’s ground-breaking scientific breakthroughs and advocacy for policy change. Featuring: Medical spotlight: Professor Tim Cox, University of Cambridge, discusses the evolution of LSD classification from initial clinical descriptions to the modern genomic era. Advocacy and leadership: Bob Stevens, CEO, MPS Society, shares his decades-long journey from a father seeking answers for his sons to a leader transforming lives for the MPS community. Childhood dementia: Megan Maack, Childhood Dementia Initiative, explains the urgent need for a unified response to paediatric neurodegeneration and the collective burden of these conditions. Newborn screening: Toni Mathieson, CEO, NPUK, makes a powerful case for expanding the UK's newborn screening programme to end the "diagnostic odyssey" for Niemann-Pick and other lysosomal storage disorders. Patient engagement: Azafaros experts Gisela Linthorst and Dominique Nijkamp explore ethical clinical trial continuity and the co-design of "humanly feasible" trials with patient families. Global access: Dr. Stefano Portolano (CEO, Azafaros) discusses the "plausible mechanism" framework and removing logistical barriers to ensure equitable global access to therapies. Mental health support: Raregivers announces a new partnership with Crisis Text Line, offering free, 24/7 confidential support to the rare disease community (Text RARE to 741741). Plus lots more news and reviews from your community.