US National Registry for Pulmonary Alveolar Proteinosis (PAP) Patients Critical to Success of Ongoing Research
Successful completion of the Multicenter International Lymphangioleiomyomatosis (LAM) Efficacy and Safety of Sirolimus (MILES) trial was critically dependent on communication and collaboration with the LAM patient community, which was made possible through their advocacy group, the LAM Foundation. “In preparation for the upcoming treatment trials for PAP, it’s important that we similarly engage the pulmonary alveolar proteinosis (PAP) patient community,” says Bruce Trapnell, MD, Professor of Medicine and Pediatrics. “Clinical investigators and patients with rare diseases need a conduit to communicate about research results and clinical studies, to answer patient questions, and provide patients a ‘seat at the table’ in planning future research studies. To this end, we are reaching out to patients and pulmonologists alike online and will be sending letters and emails to every academic center in the country and all the patients in whom we have diagnosed PAP through our prior clinical research studies.”
Interested patients can find out more and receive further information about the clinical studies for PAP and other rare lung diseases through the Contact Registry of the National Institutes of Health (NIH) Rare Diseases Clinical Research Network accessible via the internet at https://www.rarediseasesnetwork.org/registry/.
“The involvement of patients and their doctors is important to the successful development of novel diagnostics and therapies for patients with rare lung diseases,” says Trapnell.
To learn more or get involved, contact the Rare Lung Diseases Consortium Program Coordinator at 513-636-6361.Leave a reply →