PCD Foundation

10/20/2026

We are excited to introduce the PCD Foundation Network for Clinical Trials (NCT)!

Clinical trials for PCD require specialized expertise, research capacity, and the ability to coordinate research across multiple sites. The NCT brings these pieces together to support the efficient and effective development of clinical trials for PCD and help move promising research towards new treatments and, ultimately, a cure.

The NCT is a coordinated network of PCD researchers and clinical centers across the United States and Canada. The University of North Carolina at Chapel Hill serves as the Coordinating Center, working with three Regional Research Hubs:

-University of Washington and Seattle Children’s Hospital
-Children’s Hospital Colorado and National Jewish Health
-Vanderbilt University and Indiana University

Five additional Affiliate Research Hubs extend the network’s reach: Children’s Hospital of Philadelphia, Washington University in St. Louis, McGill University, the University of Toronto, and the University of British Columbia.

Through shared expertise, centralized coordination, and PCD-specific resources – including the PCDF Research Registry – the network aims to:

-Accelerate clinical trials that can change the lives of people with PCD
-Train the next generation of physicians and scientists in PCD research
-Launch new discoveries through PCD research, including PCD-specific treatments

The NCT represents years of planning and partnership across the PCD community, and creates a foundation that can continue to grow as new research and treatment opportunities emerge.

Learn more about the PCD Foundation’s Network for Clinical Trials: https://www.pcdfoundation.org/the-pcd-foundation-network-for-clinical-trials/

10/19/2026

Research helps us to better understand PCD. Clinical trials help us explore what comes next.

Today, there are no treatments available that specifically address the underlying causes of PCD. Clinical trials are an important step towards changing this – they allow researchers to evaluate potential treatments, learn whether they are safe and effective, and understand how they might improve the lives of people with PCD. In a disease with significant unmet needs, clinical trials offer hope for new and improved options for PCD treatment.

Clinical trials for a rare disease like PCD come with unique challenges. Given its rarity, PCD patients are widely dispersed and few at any single center. Finding and enrolling enough patients, coordinating across PCD clinical centers, and bringing together specialized expertise all take planning and collaboration.

This week, we’re exploring how clinical trials can help advance PCD treatments – and how the PCD Foundation supports progress through our Network for Clinical Trials.

https://www.youtube.com/watch?v=0dQB1qMM7T0

Learn about clinical trials

10/16/2026

Research is how the PCD community turns unanswered questions into better diagnosis, better care, and, ultimately, better treatments.

For a rare disease like PCD, progress depends on more than one study, one clinic, or one breakthrough. It requires the right infrastructure: connected centers, shared data, prepared trial sites, engaged patients, and trusted channels for bringing researchers, clinicians, industry partners, and families together.

That is where the PCD Foundation is focused. Through programs such as the PCD Foundation Clinical and Research Centers Network and PCDF Registries, we are helping build the foundation needed to accelerate research: expert care centers, registry participation, common research priorities, and pathways that make it easier to connect the right studies with the right patients.

Patients are essential to this work, and participation can take many forms. Some patients may start by registering in PCDF Connect to passively demonstrate community engagement and help show the size and readiness of the PCD community. Others may choose to share clinical data through the PCD Foundation Research Registry, helping researchers better understand the natural history of PCD, identify care gaps, and prepare for future studies. Others may participate more actively by completing surveys, joining observational studies, or enrolling in clinical trials when the right opportunity is available.

Every level of engagement matters. A registry entry can help demonstrate trial readiness. A clinical data point can help researchers see patterns across the community. A survey response can make lived experience visible. A clinical trial participant can help determine whether a potential treatment is safe and effective. Together, these contributions help create the evidence base needed to move from hope to action.

The PCD Foundation’s goal is to make participation more possible, more coordinated, and more meaningful—so that patients and families are not just invited into research, but recognized as essential partners in building the future of PCD care.

If you are part of the PCD community, consider taking the next step that is right for you: join PCDF Connect, ask your care team about the PCD Foundation Research Registry, respond to surveys, or learn whether a clinical trial may be a fit. Research is not something that happens to the PCD community—it happens because of it.

More on the PCD Foundation’s research efforts:

- PCDF Clinical and Research Centers Network: https://www.pcdfoundation.org/what-are-clinical-centers/
- Find a PCDF Clinical Center: https://www.pcdfoundation.org/find-a-center/
- PCDF Connect Registry: https://www.pcdfoundation.org/announcing-an-addition-to-the-pcd-foundation-registry/
- PCDF Research Registry: https://www.pcdfoundation.org/registry/
- Other activities: https://www.pcdfoundation.org/healthcare-professionals/research/

10/15/2026

The pace of PCD research has never been faster, fueled by patient participation in registries and strong international collaboration. In 2026 alone, more than 100 PubMed studies have referenced PCD. Because PCD is rare, progress increasingly depends on pooling data across the global community to reveal patterns that would be difficult to see in one center or country alone. The Global PCD Conference highlighted growing participation in PCD care networks and registries – such as the PCDF Research Registry.

This collaborative approach is also reinforcing the importance of lived experience. This year, we’ve been encouraged by an increasing research focus on understanding the social and mental impacts of living with PCD. Studies examining the lived experience, the transition from pediatric to adult care, and health-related quality of life indicators demonstrate that we’re making some progress understanding the entire scope of PCD.

We enthusiastically participate in these efforts and look forward to supporting the expansion of this critical work.

Every patient who joins a registry, completes a survey, shares their experience, or contributes to research advances understanding of PCD and accelerates the work to treat it. These contributions may feel small, but they are fundamental. They help shape research priorities, identify care gaps, support trial readiness, and move the field toward more consistent standards of care.

This October, we celebrate not only the science, but the community that makes it possible. By convening patients, researchers, and clinicians, the PCD Foundation hopes to turn individual participation into collective progress. Join us by asking your care team about registries, surveys, or studies that need patient and family input.

- More on lived experience research: https://onlinelibrary.wiley.com/doi/10.1111/hex.70316
- More on the transition from pediatric to adult care: https://pmc.ncbi.nlm.nih.gov/articles/PMC13470829/
- More on health-related quality of life indicators: https://pmc.ncbi.nlm.nih.gov/articles/PMC13469980/

10/14/2026

Research is opening new conversations about how PCD may be treated in the future.

Some of the most exciting recent PCD research is focused on the root, genetic cause of disease. A recent example is the presentation of data on RCT1100, an inhaled mRNA therapy developed by ReCode and under investigation for people with PCD from variants in the DNAI1 gene, at the Global PCD Conference in Montreal. This study marks the first time an intervention has been targeted to correct the genetic cause of PCD, rather than just treating symptoms. RCT1100 development remains in an early stage, but is an exciting step toward mutation-gene-specific approaches that may one day target an underlying cause of PCD rather than only managing symptoms.

Read more about the RCT1100 study here: https://doi.org/10.1093/ajrccm/aamag286.107

Symptom-based, non-PCD-specific therapeutics are also in development, including drugs targeting the inflammatory effects seen in bronchiectasis. Bronchiectasis is a common finding in PCD and related disorders. It describes structural damage from chronic airway infection and inflammation. Bronchiectasis is a major focus of PCD treatment and care, but it should not be mistaken for PCD. It occurs in PCD as the result of the underlying ciliary defect which is the primary problem in PCD.

The recent approval of Brinsupri™, (generic name brensocatib) a novel anti-flammatory drug developed by Insmed that has been approved by the FDA for individuals 12 and over with bronchiectasis (from any cause, including PCD) offers a new target for treating symptoms.

Read more about the ASPEN/brensocatib study here: https://www.nejm.org/doi/full/10.1056/NEJMoa2411664

These studies remind us that progress is not instant. PCD research happens because PCD patients choose to participate—even when benefits are uncertain, visits are time-consuming, or the research is still early. By signing up for the PCDF Connect Registry or the PCDR Research Registry, you help our community capture data and show we are ready to own our future.

But data is only part of the equation. Turning research into treatment opportunities takes sustained investment into clinical trial infrastructure, partnerships, and resources needed to move discoveries forward.

This Friday, October 16, we’ll gather in Chicago for An Evening to Ignite Hope to help make that progress possible. If you can’t be there in person, we still need you with us.

We invite you to join us in supporting the work that will move PCD research forward. Your gift can help build coordinated infrastructure to expand access to clinical trials – turning growing research and industry interest into real opportunities for people with PCD.

Every action helps build a future where better treatments – and ultimately, a cure – are within reach. The future of PCD research is something we can help shape together.

10/13/2026

Many PCD patients are not diagnosed until later in life, enduring a medical odyssey before they finally receive the care they need. Does early diagnosis change outcomes for people with PCD?

A 2026 study of infancy-diagnosed patients helped answer that question by following children identified very early in life. Even with early diagnosis and care, many children still experienced respiratory flare-ups, emergency visits, hospitalizations, frequent clinic appointments, and needed antibiotics during the first two years of life.

The same research found abnormal hearing in most tested children by age 2, often linked to fluid buildup, and identified early growth of Pseudomonas aeruginosa in some young children. These findings point to the importance of routinely checking hearing health, routine airway cultures, and coordinated care. They also underscore the unmet need for treatments that address the root cause of PCD—not just its symptoms, while providing clinical baselines and justification for trials involving genetic therapies.

This research exists because children and families participated. We are grateful to the families who said yes to follow-up, testing, data collection, and the work of helping researchers understand early life with PCD. Their participation helps clinicians improve care for the next child diagnosed.

Read more about the research here: https://doi.org/10.1002/ppul.71658

10/12/2026

October is PCD Awareness Month—a time to shine a light on the research moving our global community forward.

PCD research has advanced significantly—from observational data and generic lung therapies toward precision genetics and target-specific treatments. Recent developments span landmark clinical trials, new diagnostic guidance, and genetic breakthroughs. However, despite progress, there are still no approved PCD-specific therapies, and more research is desperately needed. Without it, PCD will continue to rely on therapies 'borrowed' from other disorders.

Earlier, more accurate, and accessible diagnosis remains one of the biggest opportunities to advance research and improve outcomes for people with PCD. Improved diagnostics are the first step in the broader research trajectory: they can deepen understanding of the disease, support more effective treatments, and eventually enable genetic therapies. They can also connect patients and families sooner to care, education, counseling, specialty referrals, and research opportunities that may shape future solutions.

This year, we celebrate the new ERS/ATS diagnostic guideline—the first international joint clinical practice guideline for PCD diagnosis. It recommends diagnosis through genetic testing and/or transmission electron microscopy, with tools such as nasal nitric oxide, high-speed video microscopy, and immunofluorescence providing additional support.

While these tools are all valuable, no single test is perfect, and the quality and technique of each test is critical. Whenever possible, the PCD Foundation recommends referral to an expert center.

We are grateful to every patient and family who has contributed their diagnostic experience and data to the research community. If you are part of the PCD community but haven’t received a complete diagnosis, please consider asking your care team about opportunities to put the new joint guidelines into action. And join us at the PCDF Connect Registry to stay up to date as diagnostic research continues.

Find the joint ERS/ATS diagnostic guidelines here: https://pubmed.ncbi.nlm.nih.gov/41005984/
Sign up for PCDF Connect here: https://www.pcdfoundation.org/pcdfconnect/

10/09/2026

Throughout the first week of PCD Awareness Month, we’ve been talking about the importance of patient participation in PCD research. PCDF Connect is a new registry that gives patients and families an entry point to get involved, make their voices heard, and stay up to date on PCD news and events.

Who can join?

PCDF Connect is open to individuals with PCD, whether or not they are already enrolled in the PCD Foundation Research Registry and regardless of where they receive care.

Why participate?

Patients bring essential experiences, perspectives, questions, and voices to PCD research. Your participation can help researchers better understand PCD, identify areas where more progress is needed, and learn what matters most to the patient community.

Want to learn more?

Check out this week’s webinar, The Power of Patients: PCDF Registries, to hear more about both registries: https://youtu.be/9TB5ELi2x74?si=lIXokO6Eb0zGa-SQ

Whether you are already receiving specialized PCD care or are just beginning your journey, there is a place for you in the PCD community. While we celebrate having over 1,000 people in our Research Registry, we know that there are 30,000 more PCD patients in the United States that we haven't heard from. Help us close that gap by taking a few minutes to join PCDF Connect today: https://redcap.vumc.org/surveys/?s=7YXRX7JHDHFMAYJ7

You can view our guide to medical research in PCD here:https://www.pcdfoundation.org/wp-content/uploads/2026/07/A-Guide-to-Medical-Research-PCD.pdf

10/08/2026

Two Registries, One Goal: PCD Foundation Research Registry and PCDF Connect

In keeping with the PCDF goal of a ‘registry for everyone with PCD’ we have two opportunities for you to make sure your PCD experience is counted. If you joined us for yesterday's webinar, you heard more about how these two registries work and the different ways patients can get involved. You also learned that participating in one doesn’t mean you can’t sign up for the other!

These programs represent an important investment in the PCD community and in the future of PCD research. Philanthropic support makes it possible to build and maintain the technology, staff, and resources needed to operate these registries and support the individuals with PCD who participate. Your generosity helps make these programs possible.

If you missed us, or want to revisit information shared, you can now watch the recording of the webinar. Catch up on everything here: https://youtu.be/9TB5ELi2x74?si=lIXokO6Eb0zGa-SQ

You can also read more about our registries on our website:

- PCDF Research Registry -- https://www.pcdfoundation.org/registry/
- PCDF Connect -- https://www.pcdfoundation.org/pcdfconnect/
- Support the work that makes these programs possible: https://www.pcdfoundation.org/donate/

Your participation matters. Your support matters, too. Please consider supporting these programs and sharing what you’ve learned with others impacted by PCD in your community.

10/07/2026

TODAY: Join us at 12:00 PM Eastern time for our webinar on The Power of Patients: PCDF Registries!

Have you ever wondered what participating in a registry really means, or how registries contribute to research? Dr. Michael O’Connor, Michele Manion, and Sigrid Almeida, CRC, will walk us through the details: how PCDF's Connect and Research Registries work, the differences between them, why they’re important, and how they can benefit you.

We look forward to welcoming you!

Register here: https://us02web.zoom.us/webinar/register/WN_BPcK5ZKGR_2QLMrehZNZaA #/registration