Cystic fibrosis
Synonyms: CF | Mucoviscidosis
A rare genetic pulmonary disorder characterized by sweat thick mucus secretions causing multisystem disease chronic infections of the lungs bulky diarrhea and short stature.
Newly diagnosed with
Cystic fibrosis?
Our RARE Concierge Services Guides are available to assist you by providing information, resources and connections as you navigate your rare disease journey.
Advocacy Organizations
National Organization of African Americans with Cystic Fibrosis
The mission of NOAACF is to engage, educate, and raise CF awareness in the African-American community to help bring valuable resources, knowledge, empowerment, and support to CF patients, families, healthcare professionals, and the community. Through widespread involvement, partnerships, and outreach, NOAACF's vision is to ensure that the Cystic Fibrosis diverse community is educated, informed, and made aware of CF's existence, prevalence, and impact on underrepresented communities. NOAACF's goals are to connect, engage, and raise CF Awareness in the African-American Community and beyond through its national platform and to continue individually, collectively, and synergistically raising cystic fibrosis engagement in the African American community.
CF Get Loud
To fight for patients across Canada and help them gain access to rare disease medicine. CF Get Loud made history when we removed federal roadblocks preventing CF patients the ability to access Trikafta.
Cystic Fibrosis Research Institute
To be a global resource for the cystic fibrosis community while pursuing a cure through research, education, advocacy, and support.
The Bonnell Foundation: Living with cystic fibrosis
Mission Statement: To give emotional and/or financial support to parents who have a loved one with cystic fibrosis. The Foundation also education the community so more people make Our hope is one day no CF parent will experience the pain of losing a child to this disease. Live – Breathe – Inspire
Breathe Bravely
Giving voice to cystic fibrosis
Emily’s Entourage
Emily’s Entourage (EE) accelerates research and drug development for the final 10% of people with cystic fibrosis (CF), including those with nonsense mutations. By providing critical leadership and coordination, EE drives high-impact research, cultivates multi-stakeholder collaboration, and facilitates information exchange to speed breakthroughs.
The Assistance Fund
The Assistance Fund's mission is to provide underinsured people living with life-threatening, chronic, and rare diseases access to critical treatment through financial assistance, education, and advocacy.
Cystic Fibrosis Reproductive and Sexual Health Collaborative
Our mission is to complete patient-engaged research through partnerships with people with CF, researchers, and advocates. Our research will respond to the health needs of the CF community, provide data for healthcare professionals, and ultimately pave the way for improved reproductive and sexual health resources, healthcare, and knowledge for people with CF.
Cystic Fibrosis Reproductive and Sexual Health Collaborative
Our mission is to complete patient-engaged research through partnerships with people with CF, researchers, and advocates. Our research will respond to the health needs of the CF community, provide data for healthcare professionals, and ultimately pave the way for improved reproductive and sexual health resources, healthcare, and knowledge for people with CF.
Global Liver Institute
To improve the lives of individuals and families impacted by liver disease through promoting innovation, encouraging collaboration, and scaling optimal approaches to help eradicate liver diseases.
Help Hope Live
Help Hope Live supports community-based fundraising for people with unmet medical expenses and related costs due to organ transplants or catastrophic injuries or illnesses. For 38 years, Help Hope Live has been showing clients and families how to bring together a network of relatives, friends, and neighbors in fundraising efforts to help cover the cost of uncovered medical expenses. These efforts play a critical role in helping our clients recover and maintain their health and independence. Since 1983, we have helped thousands of people raise millions of dollars for Help Hope Live to pay a wide range of expenses, including out-of-pocket costs for: medications, durable medical equipment, home health care, wheelchair-accessibility modifications, physical therapy, innovative treatments, medical travel and temporary relocation, even emergency living assistance. Our program has also helped thousands pay it forward and assist others with their medical expenses. Annually, we help place medical care within reach of about 5,000 families across the nation.
Syndromes Without A Name (SWAN) Australia
Provide information, support and advocacy to families caring for a child with an undiagnosed or rare genetic condition.
Center for Chronic Illness
The Center for Chronic Illness (CCI) promotes well-being and decreases isolation for those impacted by ongoing health challenges through support and education.
Inclusive Skating
TO INCLUDE, INFORM AND INSPIRE people around the globe through the joy of skating, reflecting the compassion, respect and innovation that make ours the world’s premier inclusive organisation. Inclusive Skating is dedicated to the advancement of public participation in sport and the promotion of equality and diversity. Our primary objective is the development and implementation of programming which fosters the inclusion of skaters with any form of impairment or disability. Our ethos of inclusion extends to skaters of all ages and backgrounds; we welcome all with open arms. Values of empathy, integrity and empowerment are central to the pursuit of these goals. We believe that everyone should have the opportunity to experience the community, camaraderie, self-fulfilment, achievement, challenge, and thrill that participation in skating sports offers. We work to create these opportunities by offering activities, educational resources, events, training, and championships for our community.
Rare Disease Ghana Initiative
To improve the wellbeing and quality of life of persons living with undiagnosed and rare diseases in Ghana
Our Odyssey
Connecting young adults impacted by a rare or chronic condition with social and emotional support in the hope of improving their quality of life.
Jamal’s Helping Hands
Jamal's Helping Hands provides services for patients affected by rare disease and their families. With a vision to become a premier resource and national leader in education, outreach, advocacy, and support to individuals affected by rare disease. JHH enhances the quality of its clients lives by providing an array of services to make the experience of rare disease easier.
Patient Advocate Foundation
Patient Advocate Foundation (PAF), founded in 1996, is the nation’s leading direct patient services organization whose mission is to safeguard patients with chronic, life-threatening, and debilitating diseases through effective mediation of issues related to access to care and preservation of financial stability. Patients must be seeking help with any of the following needs related to their diagnosis: • Accessing prescribed healthcare, including understanding their healthcare benefits • Issues related to their health insurance benefits, including denials of care, out-of-pocket costs, network issues, and insurance options • Financial concerns, including cost-of-living expenses and medical expenses • Practical needs, including transportation and nutritional needs • Assistance with filing an application for disability Since its inception, PAF has provided direct, sustained patient assistance to more than 1.7 million patients and touched many more through its website and outreach events. Amongst the more common issues that patients and caregivers call PAF for help with include the inability to afford transportation expenses, inability to afford rent/mortgage, inability to afford their co-pay for medications, and inability to afford utilities and resulting shut-off notices. PAF serves patients with numerous health conditions, the majority of which have some form of cancer. Non-cancer diagnoses included rare diseases, chronic and debilitating conditions, nervous system conditions, cardiovascular conditions and vascular disease, autoimmune diseases, and diabetes. PAF solicits and receives donations to its programs from a multitude of sources including government agencies, non-profit organizations and for-profit organizations in the healthcare sector, including pharmaceutical manufacturers and healthcare providers. Patient Advocate Foundation also has several distinct programs targeting specific populations with an emphasis on the underserved.
Clinical Trials
For a list of clinical trials in this disease area, please click here.