Based at Sanford Research, a nonprofit research institution, CoRDS is a centralized international patient registry for all rare diseases.
The Healthcare Navigation Project INC is a comprehensive life skills program which helps to bridge the healthcare gap and create healthcare equity, by providing support and education to navigate the complexities of the healthcare system. They empower underserved communities, individuals with chronic diseases, and those with educational disabilities to access essential healthcare, mental health services,
The Cystic Fibrosis Engagement Network (CFEN) serves as a leading educational and advocacy organization focused on policy matters impacting cystic fibrosis patient access to optimal care. CFEN is made up of member organizations from across the United States. Together, we hope to affect real change for the CF community. Click the CFEN logo to get
Emily’s Entourage is an innovative 501(c)3 that accelerates research for people in the final 10% of the cystic fibrosis (CF) population that do not benefit from existing CFTR modulators. Since 2011, Emily’s Entourage has awarded millions of dollars in research grants, launched a now-acquired CF gene therapy company, developed a patient registry and clinical trial
First Wave BioPharma, Inc. is a clinical stage biopharmaceutical company specializing in the development of targeted, non-systemic therapies for gastrointestinal (GI) diseases. First Wave is currently advancing a therapeutic development pipeline populated with multiple clinical stage programs built around its two proprietary technologies – the biologic adrulipase, a recombinant lipase enzyme designed to enable the
Alcresta develops rare disease, enzyme-based products helping to address fat malabsorption in patients who are enterally fed at home or in the hospital. Our RELiZORB® (immobilized lipase) is indicated for use in pediatric patients (ages 5+) and adult patients to hydrolyze fats in enteral formula. Please see Instructions for Use at wwww.relizorb.com.
4DMT is a clinical stage biotherapeutics company harnessing the power of directed evolution for genetic medicines targeting large market diseases. 4DMT seeks to unlock the full potential of genetic medicines using its proprietary invention platform, Therapeutic Vector Evolution, which combines the power of the Nobel Prize-winning technology, directed evolution, with approximately one billion synthetic AAV