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Alliance Against HMERF

Our Progess

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We continue to make progress.

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Christine Duane

CEO and Casey’s Mom

Our roadmap to life-saving treatments

A life-saving answer to HMERF is within our reach, and we are making great strides. Because of you, we can make a difference and continue our relentless pursuit of developing a therapeutic for HMERF.

STAGE 1

Target Identification

For HMERF the genetic defect is located on titin protein chain at exon 344. These mutations can disrupt the normal folding and function of the titin protein , leading to muscle damage and respiratory issues.

COMPLETE

STAGE 2

Develop Model

Researchers study the target protein to understand its functionality and then synthesize potential drug molecules that might interact with the target protein. Thousands of compounds are analyzed, seeking ones with therapeutic value..

COMPLETE

STAGE 3

Preclinical Testing

This stage can take up to 6–7 years as researchers synthesize and purify the drug, conduct limited animal testing, and evaluate the therapy in lab (in vitro) and animal (in vivo) models for safety, efficacy, and how it’s processed in the body.

5TH YEAR

STAGE 4

Clinical Trials

This phase tests the drug in humans across three stages. Phase 1 focuses on safety and metabolism in healthy volunteers. Phase 2 assesses safety, effectiveness, and dosage in patients with the target condition. Phase 3 is the largest and costliest, confirming safety and effectiveness in a broader group. Afterward, researchers file a New Drug Application (NDA) with the FDA for review and expert advisory input.

NEXT STEP

STAGE 5

Take drug to market

Using the groundwork of the previous phases, the researchers work toward approval and commercial production of the target treatment that paves the way to changing the lives of these rare disease patients.

FINAL STEP