The National Niemann-Pick Disease Foundation (NNPDF) is at the forefront of advancing research and supporting efforts to discover effective treatments and a cure for Niemann-Pick Disease. These rare genetic conditions, encompassing Acid Sphingomyelinase Deficiency (ASMD) and Niemann-Pick type C (NPC), affect the body’s ability to metabolize lipids, leading to progressive and often debilitating symptoms. NNPDF’s work has included funding innovative research, fostering collaboration among scientists, and advocating for the patient voice and perspective be at the center of therapeutic development of life-changing therapies.

Recent progress has brought significant hope to the Niemann-Pick community with the approval of groundbreaking treatments. Xenpozyme® (Olipudase Alfa) is approved for the treatment of Acid Sphingomyelinase Deficiency (ASMD), providing a much-needed option for managing this disease. Additionally, therapies including Aqneursa (IB1001) and Miplyffa (arimoclomol) are approved to address critical aspects of Niemann-Pick Disease type C, offering new avenues for care and management. These advancements highlight the importance of NNPDF’s funding and advocacy efforts in driving research from the lab to the clinic.

NNPDF’s dedication extends beyond treatment approvals, as the foundation continues to support clinical trials and patient registries that pave the way for future breakthroughs. By working closely with families, researchers, and industry leaders, NNPDF is creating a brighter future for those affected by Niemann-Pick Disease, ensuring that no family faces this journey alone.