Engage Health Inc.

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.

11/13/2025

Exciting news! Patti A. Engel, R.N., B.S.N., CEO & President of Engage Health, will join IFOPA Family Services Manager Hope Newport this Saturday, November 15, at the 2025 IFOPA Family Gathering to present key findings from the Health Insurance Literacy and Burden of Access Study.

We’re so grateful to the individuals and families who made this research possible by sharing their experiences and insights. International FOP Association (IFOPA)

10/27/2025

We were proud to partner with SmithSolve and the American Neuromuscular Foundation on the Late-Onset Neuromuscular Disease Consortium (LONDC) on this study. To review the full study, link here: https://tinyurl.com/4s8m5skj

Last week at , SmithSolve’s EVP Katie Burns spotlighted the urgent need for better recognition and referral pathways for Late-Onset Neuromuscular Diseases ( ).

Key findings from our research with more than 120 physicians:
🔹 Low awareness of LONDs
🔹 Referrals to specialists are rare
🔹 Multiple barriers slow timely diagnosis
🔹 Decision-support tools are urgently needed

We’re proud to partner with the American Neuromuscular Foundation on the Late-Onset Neuromuscular Disease Consortium ( ) to advance education, tools, and resources that improve LOND detection and patient care.

Get involved or learn more at https://LONDC.neuromuscularfoundation.org

10/22/2025

It was a great honor to present the findings from our Health Insurance Literacy study in DM1 at . We’re grateful to our collaborators, Dyne Therapeutics and the Myotonic Dystrophy Foundation, for their partnership. To view the poster, click here: https://tinyurl.com/3wyeadvy

Proud to partner with Dyne Therapeutics and Engage Health Inc. at 💙 Together we shared early findings from our Health Insurance Literacy in Type 1 (DM1) study—highlighting how collaboration helps move research forward and strengthens the community.

10/15/2025

We’re looking forward to joining the conversation at the NORD Rare Diseases & Orphan Products Breakthrough Summit®, taking place October 19–21 in Washington, D.C.!

This year, our team will be presenting a poster developed in collaboration with Dyne Therapeutics and the Myotonic Dystrophy Foundation (MDF). “Health Insurance Literacy in Myotonic Dystrophy Type 1 (DM1): An Interim Report” explores the challenges of navigating healthcare coverage and the opportunities to improve access as new treatments emerge.

If you’ll be attending the , we’d love to connect with you. To schedule a time to meet, contact Anne at amelchior@engagehealth.com
or call 651-994-0510.

10/09/2025

Some of the most meaningful insights begin with families willing to share their stories.

We’re proud to have contributed to this study, presented by Stacy Pike-Langenfeld at the APHL Newborn Screening Conference, which may help establish a biomarker to support faster diagnosis for children with Krabbe disease. KrabbeConnect Rosenau Family Research Foundation

Stacy Pike-Langenfeld presented at the Association of Public Health Laboratories (APHL) Newborn Screening Conference - Characterizing Irritability in : Implications for Early Diagnosis and Treatment Decisions

What we found:
-Krabbe irritability differs from colic in tone, associated behaviors, frequency, and prevelance
-Collaboration with experts and AI tools to analyze cries may help detect distinct patterns
-These patterns could help serve as a biomarker, supporting faster diagnosis
-This approach is especially valuable in regions without NBS for Krabbe disease

*We are immensely grateful to the families that participated - KrabbeConnect will work to publish this data.

* This work wouldn’t be possible without the funding of Rosenau Family Research Foundation

* We are thankful for the recruitment help from United Leukodystrophy Foundation and Partners for Krabbe Research

10/07/2025

The APHL Newborn Screening Conference kicked off yesterday and Avery Coupe’s story was shared by her mom, Lindsey Coupe. Avery was diagnosed with Krabbe disease and some of the powerful statements made by Lindsey were as follows:

“Avery was born with this silent disease until symptoms and then our options were gone for treatment - our only choice was to give her the best quality of life we could until she passed.”

“Avery had lots of adventures - as much as we could squeeze in- however lots of doctors appointments, medical equipment, OT, PT, early intervention - and then we went from a 2 income family to a 1 income family.”

”I hope Avery’s story is the last one I see, it might be the only Krabbe story in Rhode Island, but I hope it’s the last story without access to treatment.”

10/01/2025

Engage Health was honored to partner with KrabbeConnect with generous support from the Rosenau Family Research Foundation, on this important study — now being presented as a poster at the APHL Newborn Screening Conference.

09/30/2025

We’re excited to announce that Patti and Anne will be attending the NORD Rare Diseases & Orphan Products Breakthrough Summit®, taking place October 19–21, 2025, in Washington, D.C.

They would welcome the opportunity to meet with you during the event. To schedule an appointment, please contact Anne at amelchior@engagehealth.com or call 651-994-0510. National Organization for Rare Disorders, Inc. (NORD)

09/17/2025

Today, September 17, Regeneron Pharmaceuticals has released results from its OPTIMA Phase 3 trial of the drug garetosmab.

THANK YOU to the 63 FOP community members who participated in this trial!

You can read the press release at ifopa.org/regeneron-optima-trial-update

This press release does NOT mean that the drug being tested in this trial, garetosmab, is available for doctors to prescribe. This press release provides a first look at the trial results. Regeneron will report the full trial results to the U.S. Food and Drug Administration and then make decisions about its next steps.

Important Note: The OPTIMA clinical trial is active, but not currently recruiting, so it is not accepting any new patients.

Questions? Email us at together@ifopa.org

09/15/2025

Engage Health is honored to be a Silver Level Sponsor of WORLDFair™ 2025! 🌟We’re proud to support this important gathering that unites patients, caregivers, clinicians, researchers, and industry leaders to advance care in rare lysosomal storage diseases.
📅 Date: September 19, 2025
📍 Location: Minnesota Landscape Arboretum + Virtual (Zoom)
⏰ Time: 10 AM – 2 PM CST
Event Highlights:
✅ Plenary session with the latest updates in lysosomal storage disorders
✅ Breakout sessions on Fabry, Gaucher, Pompe, MPS, and Cystinosis
✅ Exhibits from patient groups, academic centers, and leading biopharma partners
This hybrid event is a one-of-a-kind opportunity to connect, learn, and collaborate across the rare disease community. 🔗 Register here: z.umn.edu/worldfair25