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?
Pharma & Biotech
Pharma & Biotech
Tenacia’s ZTALMY launch shows new blueprint for orphan neurology drugs in China
ZTALMY gains commercial launch in China for CDKL5 seizures. Find out what this means for rare disease drug access and neurology strategy.
Pharma & Biotech
Is givinostat’s entry into Japan a turning point for DMD care and for JCR Pharmaceuticals’ rare disease strategy?
Find out how Italfarmaco and JCR are bringing the DMD drug givinostat to Japan and what it means for rare disease innovation in Asia.
Pharma & Biotech
Acadia expands DAYBUE delivery formats for Rett syndrome with powder-based STIX
Acadia Pharmaceuticals has announced U.S. Food and Drug Administration approval for DAYBUE STIX (trofinetide) for oral solution, a new powder-based formulation of its existing drug DAYBUE used in the treatment of Rett syndrome. The approval covers use in adult and pediatric patients two years and older and is based on bioequivalence data, not new clinical
Pharma & Biotech
What Orphalan’s Orphelia Pharma acquisition reveals about the next phase of rare pediatric drug consolidation
Orphalan, the international pharmaceutical company known for developing orphan drugs, has announced the acquisition of Orphelia Pharma, a specialist in rare and severe pediatric diseases. The deal enhances Orphalan’s position in the European rare disease market, particularly within pediatric neurology and oncology, and signals a strategic pivot toward deeper vertical integration in pediatric formulations and
Pharma & Biotech
Could utrophin become the next frontier in DMD care? Inside Ractigen’s RNAa push
Ractigen Therapeutics has initiated human testing of RAG-18, a novel small activating RNA (saRNA) therapeutic candidate for Duchenne muscular dystrophy (DMD), by dosing the first patient in an investigator-initiated trial at Peking Union Medical College Hospital in Beijing. The study is being led by Professor Dai Yi, a recognized expert in neuromuscular disorders, and will