Engage Health Inc.

02/10/2026

We were excited to present feasibility results from the Living with Cystinosis as an Adult: Community Insights Study at the 2026 WORLDSymposium last week!
A huge thank you to Recordati and Thiogenesis Therapeutics for their support, and to our partners at Next Generation of Cystinosis for helping advance research centered on adult lived experience.

02/05/2026

We are proud to be collaborating with Dr. Jeanine Jarnes and Next Generation of Cystinosis on Living with Cystinosis as an Adult: The Community Insights Study, which will be presented today by our Program and Operations Director, Austin Letcher, from 3:30–5:30 PM PT at the WORLDSymposium (Poster 224 – Kiosk 26-A).

This research centers on elevating the lived experiences of adults with cystinosis—capturing emotional, psychosocial, and quality-of-life insights from adults which is underrepresented in medical literature. By amplifying these perspectives, this work aims to increase awareness across the scientific, medical, and patient communities to support improved understanding and care.

We are deeply grateful to everyone who has shared their voice so far and look forward to the impact this work can have for the cystinosis community.

01/27/2026

Engage Health will be participating and presenting a poster at WORLDSymposia. Reach out if you will be attending and connect with us by emailing Austin at aletcher@engagehealth.com or calling the office at 651-994-0510.

01/16/2026

We are honored to be part of this study in Krabbe disease and to support the Krabbe community. KrabbeConnect Rosenau Family Research Foundation United Leukodystrophy Foundation

🎉 EEEK — WE’RE SO EXCITED!!! 🎉

After 2 years in the making, the full article is now published: Caregiver-reported disease burden in Krabbe disease: evaluating outcomes of hematopoietic stem cell transplantation 🧬

🔗 Read the full article: https://link.springer.com/article/10.1186/s13023-025-04176-3

A HUGE shout-out to the families who shared their experiences to help shape a better future for those impacted by .

We are deeply grateful to Rosenau Family Research Foundation for funding this work, Engage Health Inc. for conducting caregiver interviews and collecting the data, and United Leukodystrophy Foundation and Partners for Krabbe Research for helping raise awareness and amplify this important study. 💜

This publication is packed with important data families, clinicians, and states can use to better understand real-world outcomes of HSCT and inform newborn screening and treatment decisions.

✨ 3 Key Highlights:
• Early HSCT is associated with reduced disease burden and better overall quality-of-life scores compared to late or no transplant.
• Caregivers of early-transplanted infants reported improvements in sleep, mental health, and family/social relationships.
• The study provides critical evidence for states evaluating Krabbe disease newborn screening programs and understanding HSCT’s impact on families.

📌 Most importantly, this work will help families newly navigating a Krabbe diagnosis make more informed decisions about hematopoietic stem cell transplantation with data grounded in caregiver experience and patient-centered outcomes.

Let’s share this far and wide — real insights that matter to our community! 💜✨

01/01/2026

Happy New Year!

12/31/2025

Honored to collaborate with Breathe California in 2025!

The strength of Breathe California’s important work and proven public health outcomes in our communities over many decades inspires organizations, government agencies, and corporations to fund and promote our programs. We are grateful for Engage Health’s support in 2025. Thank you!!!

12/29/2025

We are honored to be part of this first-of-its-kind study in Krabbe disease and to support the Krabbe community. KrabbeConnect Rosenau Family Research Foundation

Amazing News! Our publication has been accepted by Orphanet Journal of Rare Disorders. A few last steps are needed before the journal will make it available to the public.

This milestone matters because:

*40+ families courageously participated, sharing detailed insights into their journey with Krabbe disease.

*Families met virtually with Engage Health to provide robust, real-world data about the challenges and decisions they faced.

*Dr. Nicholas Bascou and Dr. Paul Orchard provided expert oversight—ensuring the data was carefully reviewed, analyzed, and categorized to the highest scientific standards.

*This is the first study of its kind in Krabbe disease to compare caregiver burden across transplant timing and disease onset.

*These findings will help families and clinicians make more informed decisions about transplant and support future research, care, and advocacy efforts.

This publication is a testament to the strength of the Krabbe community and the ongoing commitment to improving care, quality of life, and access to treatment.

This critical research required an investment of more than $200,000, generously funded through a grant from the Rosenau Family Research Foundation.

12/25/2025

Wishing everyone a warm and joyful holiday!

12/16/2025

Congratulations to Parent Project Muscular Dystrophy!

🎉 Today, we’re celebrating an exciting milestone for the community: Duchenne has been added to the Recommended Uniform Screening Panel (RUSP)!

By reducing the diagnostic delays that have affected Duchenne families for generations, early identification through can:
- Allow families to connect with care and neuromuscular experts sooner
- Access approved therapies and supportive interventions earlier
- Improve long-term health outcomes

💪This milestone is a testament to the power of collaboration and this community’s shared commitment to ensuring every child with Duchenne, regardless of zip code, has the best possible start in life. Learn more: https://www.prnewswire.com/news-releases/us-department-of-health-and-human-services-adds-duchenne-to-recommended-uniform-screening-panel-302643725.html?tc=eml_cleartime

Muscular Dystrophy Association

11/27/2025

This Thanksgiving, our hearts are full of gratitude for the rare disease community — for every patient, caregiver, and partner who contributes to the research that moves us all forward.

At Engage Health, we’re grateful for the opportunity to work alongside you -collecting data to drive actionable insights.

🦃 From our family to yours, Happy Thanksgiving!

11/17/2025

Please join us in celebrating Jiayi (Bryan) Yan, our newest Rare Disease Difference Maker®!
As an advocate and researcher living with achondroplasia, Bryan is working to expand understanding, inclusivity, and treatment options for adults in his community. His leadership reflects both compassion and extraordinary determination.
Read Bryan’s full story and join us in celebrating his contributions to the rare disease community.
https://www.engagehealth.com/rare-disease-difference-maker/jiayi-bryan-yan/

11/14/2025

Engage Health is proud to be a part of this important research publication for the Krabbe disease community. KrabbeConnect Rosenau Family Research Foundation

Sleep and mental health were the areas of life most impacted for all IKD caregivers as well as LIKD transplanted late/not. LIKD transplanted early were most impacted by sleep and relationship with the spouse/partner as well as physical health.
(Scale: 1 = “not at all” to 5 = “extreme”)

These findings come from the caregiver study conducted by KrabbeConnect and Engage Health Inc., through a grant funded by the Rosenau Family Research Foundation

A manuscript from this two-year project has been submitted for publication—we’ll keep you updated when it’s released.

💙 Thank you to all the families who participated and helped make this research possible.