New treatment for heart failure in Duchenne muscular dystrophy patients
Official title: IND-enabling safety and toxicity testing of a subcutaneous formulation of the PTP1B inhibitor MSI-1436, a novel strategy for treating fatal cardiomyopathy in Duchenne muscular dystrophy patients
Funded at Revidia Therapeutics, INC · NIH-10913659 · Data updated 2026-11-12
This study is testing a new under-the-skin treatment for heart problems caused by Duchenne muscular dystrophy (DMD) to see if it can help improve heart function and possibly help patients live longer.
Quick facts
| Grant type | Sbir 2 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Revidia Therapeutics, INC NIH-funded |
| Lab location | 1 site (Ellsworth, UNITED STATES) |
| Project start | September 20, 2024 |
| Project end | September 19, 2025 |
| Funding year | 2024 |
| Award (this year) | $1,358,179 |
| NIH project number | 1R44HL174171-01 |
| Project ID | NIH-10913659 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are children and young adults diagnosed with Duchenne muscular dystrophy who are experiencing heart failure or cardiomyopathy.
✕Less likely to help
Patients with other forms of heart failure not related to Duchenne muscular dystrophy may not benefit from this research.
What this research studies
This research focuses on developing a new subcutaneous treatment for heart failure caused by cardiomyopathy in patients with Duchenne muscular dystrophy (DMD). The approach involves a small molecule called MSI-1436, which has shown safety in humans and works by inhibiting a specific enzyme that hinders the body's natural repair processes. By reactivating these processes, the treatment aims to improve heart function and potentially extend the lives of DMD patients. The research is crucial as there are currently no approved medications specifically for DMD-related cardiomyopathy.
Why it matters
Potential benefit: If successful, this research could provide a much-needed treatment option for heart failure in DMD patients, potentially improving their quality of life and longevity.
How similar studies have gone: While there has been limited research specifically targeting DMD cardiomyopathy, the approach of using small molecules to enhance cardiac repair has shown promise in other contexts.
Where this research is happening
-
Revidia Therapeutics, INC
Ellsworth, United States Active
Researchers
- Principal investigator: Strange, Kevin — Revidia Therapeutics, INC
- Study coordinator: Strange, Kevin
Related research
Other NIH-funded projects on the same topics.
-
How immune cells affect the heart in Duchenne muscular dystrophy
This work looks at how immune cells called macrophages contribute to heart damage in people with Duchenne muscular dystrophy.
-
3D printed human heart model for Duchenne muscular dystrophy (DMD) cardiomyopathy progression
A patient-friendly 3D-printed heart model using stem cells and gene editing helps study how DMD affects the heart over time and tests potential therapies.
-
Connexin-43 and Duchenne heart problems: a new target to protect the heart in DMD
Researchers are studying whether a heart protein called Connexin-43 can be adjusted to help people with Duchenne muscular dystrophy avoid dangerous heart problems.
-
Predicting heart disease progression in Duchenne and Becker muscular dystrophy
This project will look for blood tests and heart-imaging signs that predict how heart disease will progress in people with Duchenne or Becker muscular dystrophy and in genetic carriers.
-
Investigating Connexin 43's role in heart issues related to Duchenne muscular dystrophy
This study is looking at how a protein called Connexin 43 affects heart health in people with Duchenne muscular dystrophy, with the goal of finding new ways to help prevent heart problems like arrhythmias and heart failure.
-
Understanding heart problems in Duchenne and Becker muscular dystrophy
This study is looking at heart problems that can happen in people with Duchenne and Becker muscular dystrophy to find better ways to check heart health and track how the disease changes over time, which will help improve treatments and future drug trials for those affected.
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.