Niemann-Pick Type C Disease Pipeline The therapeutic pipeline for Niemann-Pick Type C (NPC) disease is advancing to address the urgent need to slow its progressive neurological decline. Current treatments like miglustat delay symptoms but don’t target underlying cholesterol and lipid … Continue reading →

Mineral Metabolism Disorder Pipeline The therapeutic pipeline for mineral metabolism disorders is rapidly expanding, focusing on targeted hormonal modulators, novel binders, and advanced chelation to restore mineral balance and prevent complications. Current treatments manage symptoms but often fail to correct … Continue reading →

Sturge-Weber Syndrome Pipeline The therapeutic pipeline for Sturge-Weber syndrome (SWS) is progressing, focusing on targeted vascular modulators, new anti-seizure treatments, and potential gene-based therapies to tackle the root causes of this rare neurocutaneous disorder. Current management—seizure control, glaucoma treatment, and … Continue reading →

Rabies Infection Pipeline The therapeutic pipeline for rabies is steadily advancing, with new approaches aiming to enhance prevention and post-exposure care. While current PEP with vaccines and immunoglobulin is effective if given promptly, limited access, high costs, and lack of … Continue reading →

Familial Lipoprotein Lipase Deficiency Pipeline The therapeutic pipeline for familial lipoprotein lipase deficiency (LPLD) is advancing with innovations in gene therapy and lipid-modulating agents aimed at addressing the underlying genetic cause. Current treatments, like strict dietary fat restriction, help manage … Continue reading →