Mark E. Wylam, M.D.

The purpose of this study is to determine if DNA originating from the transplanted heart or lung can be detected in the blood and used to monitor for rejection.

The purpose of this study is to  identify the magnitude of variation among intensivists in use of diagnostic testing and to determine whether variation in diagnostic test ordering patterns among intensivists attributables to their personal characteristics, or to factors in the clinical environment.

This trial will consist of three arms: Part A, Part B, and Part C. Part A has two groups. The first group will enroll adult subjects with cystic fibrosis (CF) into a single ascending dose (SAD) treatment group. The second group will enroll adult subjects with CF, including those on background treatment with ORKAMBI® and those not on a cystic fibrosis transmembrane conductance regulator (CFTR) modulator, into a multiple ascending dose (MAD) treatment group. Part B will enroll adult subjects with CF currently on stable ORKAMBI® background therapy for a minimum of 3 months into a Phase II treatment group consisting of two cohorts. Part C will enroll adult subjects with CF, including those on background treatment with KALYDECO® and those not on a CFTR modulator, into a Phase II treatment group consisting of three cohorts. Approximately 136 subjects will be enrolled.

The purpose of this study is to evaluate the effectiveness of inhaled molgramostim, administered open-label, to adult cystic fibrosis subjects with chronic pulmonary nontuberculous mycobacterial (NTM) infection, with or without ongoing antimycobacterial guideline based combination therapy.

The purpose of the Cystic Fibrosis Patient Registry is to gather and maintain data on all patients with the disease so that current and accurate data can be provided to researchers and clinicians regarding practice patterns, age and gender distributions, clinical outcomes, mortality and morbidity rates, and so that epidemiologic research can be performed.

The purpose of this study is to provide evidence of the safety and effectiveness of inhaled mannitol for adult patients with cystic fibrosis.