Could Spruce Biosciences finally change the Sanfilippo syndrome type B treatment landscape?
Spruce Biosciences’ MPS IIIB data raise a pivotal question: can tralesinidase alfa turn biomarker gains into disease-changing therapy?
Oral Pompe therapy S-606001 moves to Phase 2 as Shionogi expands rare disease focus
Shionogi’s S-606001 enters global Phase 2 as the first oral GYS1 inhibitor tested as an add-on to ERT in late-onset Pompe disease. Read the analysis.
What the EU’s Elfabrio dosing update means for Fabry disease treatment burden
EU approves monthly Elfabrio dosing for Fabry disease. Discover how reduced infusion frequency could reshape enzyme therapy and treatment burden.
Can Idorsia Pharmaceuticals Ltd’s lucerastat redefine Fabry disease treatment beyond enzyme replacement therapy?
Discover how Idorsia Pharmaceuticals Ltd’s FDA-aligned Phase 3 plan could make lucerastat the first oral therapy for all Fabry disease patients. Read the analysis.
Is JCR unlocking the CNS for lysosomal storage diseases before gene therapy gets there?
Find out how JCR Pharmaceuticals is redefining lysosomal disease therapy with brain-targeted delivery platforms.