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Explore NNPDF publications to better understand Niemann-Pick disease.

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Real-life impacts of olipudase alfa: the experience of patients and families taking an enzyme replacement therapy for ASMD (February 2024)
Assessment of health state utilities associated with adult and pediatric acid sphingomyelinase deficiency, The European Journal of Health Economics (2024)
Acid sphingomyelinase deficiency: burden of disease and real-world impact of enzyme replacement therapy on pediatric patients and caregivers, WORLDSymposium poster 2023
The impact of olipudase alfa on QoL in pediatric ASMD patients with neurologic disease, WORLDSymposium poster 2023
Access burdens reported by patients vs. caregivers in acid sphingomyelinase deficiency (ASMD), WORLDSymposium poster 2023
Collaboration between patient advocacy and industry to create a master protocol to investigate acetyl-L-leucine for NPC, GM2 gangliosidoses and ataxia-telangiectasia, WORLDSymposium poster 2023
Fear related to access issues in acid sphingomyelinase deficiency (ASMD), WORLDSymposium poster 2023
Health insurance literacy in acid sphingomyelinase deficiency (ASMD), WORLDSymposium poster 2023
Health insurance literacy and burden of access in Niemann-Pick disease, Engage Health and NNPDF (September 2022)
Impact and burden of acid sphingomyelinase deficiency from a patient and caregiver perspective, Scientific Reports
Executive summary: understanding health insurance literacy and challenges in accessing health services in Niemann-Pick disease (September 2022)
Health insurance literacy and health services access barriers in Niemann-Pick disease: the patient and caregiver voice, Orphanet Journal of Rare Diseases (September 2, 2022)
Qualitative study highlights need for increased ASMD caregiver support, Patient Worthy (February 28, 2022)
A qualitative study to understand caregivers' burden of ASMD, WORLDSymposium 2022
Impacts and burden of Niemann-Pick type C: a patient and caregiver perspective, Orphanet Journal of Rare Diseases (November 24, 2021)
Impact and burden of acid sphingomyelinase deficiency from a patient and caregiver perspective, Scientific Reports (October 2021)
Challenges of access: understanding health insurance literacy and challenges in accessing health services in Niemann-Pick disease, WORLDSymposium 2021
Rare disease patient communities deserve access to data from failed clinical trials, by Joslyn Crowe
How patient advocacy groups are adapting to Covid-19, by Justin Hopkin, CheckRare (November 2020)
Invisible Threads: uncovering the purpose of community connections, Sanofi Genzyme (October 2020)
Living beyond my rare disease, by Evren Ayik, The Mighty (October 6, 2020)
Getting a treatment is not the end of the road in rare diseases, by Joslyn Crowe, HCPLive (September 2020)

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