Global Efforts Towards Developing Treatment Options for Patients With Rare LSDs

Global Efforts Towards Developing Treatment Options for Patients With Rare Lysosomal Storage Disorders by Madaline Spencer and Scott Harwood| Published on: Mar 5, 2026 Bob Stevens, Group CEO of the MPS Society and Rare Disease Research Partners in the UK, discusses global efforts towards developing treatment options for patients with rare lysosomal storage disorders (LSDs). https://checkrare.com/global-efforts-towards-developing-treatment-options-for-patients-with-rare-lysosomal-storage-disorders/

Autologous Hematopoietic Stem Cell Gene Therapy With Ex-Vivo LV Platform in MPS II

Autologous Hematopoietic Stem Cell Gene Therapy With Ex-Vivo LV Platform in MPS II by Madaline Spencer and James Radke, PhD| Published on: Mar 12, 2026 Brian Bigger, PhD, Professor of Advanced Therapeutics at the University of Edinburgh, discusses the first-in-human experience of autologous hematopoietic stem cell gene therapy (HSCGT) using a novel ex-vivo lentiviral (LV) platform to correct mucopolysaccharidosis II (MPS II). https://checkrare.com/autologous-hematopoietic-stem-cell-gene-therapy-with-ex-vivo-lv-platform-in-mps-ii/

Gene Therapy in Patients With MPS IIIA

Gene Therapy in Patients With MPS IIIA by Madaline Spencer| Published on: Mar 18, 2026 Brian Bigger, PhD, Professor of Advanced Therapeutics at the University of Edinburgh, discusses hematopoietic stem cell gene therapy (HSCGT) in patients with mucopolysaccharidosis IIIA (MPS IIIA; Sanfilippo syndrome type A). https://checkrare.com/gene-therapy-in-patients-with-mps-iiia/

Real-World Perspective on Daily Challenges of Living With MPS II

Real-World Perspective on Daily Challenges of Living With MPS II by Madaline Spencer and Joe Haddad| Published on: Mar 13, 2026 Kristin McKay, President and Executive Director of Project Alive, discusses the real-world perspective on daily challenges of living with mucopolysaccharides II (MPS II). https://checkrare.com/real-world-perspective-on-daily-challenges-of-living-with-mps-ii/

Chiesi Rare Disease Highlights at WORLDSymposia 2026

Chiesi Rare Disease Highlights at WORLDSymposia 2026 by Madaline Spencer| Published on: Mar 16, 2026 Rachele Berria, MD, PhD, Senior Vice President, Head of Global Medical Affairs at Chiesi Rare Diseases, gives an overview of the company’s highlights at WORLDSymposia 2026. https://checkrare.com/chiesi-rare-disease-highlights-at-worldsymposia-2026/

30 Years of the Fabry Support and Information Group by Madaline Spencer and Kimberly Waller| Published on: Apr 1, 2026 ++

30 Years of the Fabry Support and Information Group by Madaline Spencer and Kimberly Waller| Published on: Apr 1, 2026 https://checkrare.com/30-years-of-the-fabry-support-and-information-group/ Jeff’s Journey With Fabry Disease by Chiesi USA| Published on: Mar 31, 2026 https://checkrare.com/jeffs-journey-with-fabry-disease/

SMA in Focus: Practical Insights from MDA 2026 Key data updates, therapeutic strategies, and evolving standards of care in spinal muscular atrophy

https://checkrare.com/sma-in-focus-practical-insights-from-mda-2026/