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Gene Editing Therapy

Treatment of a Single Patient With CRD-TMH-001

Phase 1
Waitlist Available
Led By Brenda Wong, MD
Research Sponsored by Cure Rare Disease, Inc
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be between 18 and 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 year
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing a new treatment that uses advanced technology to fix faulty genes in patients with a rare form of Duchenne muscular dystrophy. The goal is to see if this treatment is safe and effective over time, with long-term follow-up. The treatment works by directly changing the DNA to correct the genetic problem causing the disease. Exon skipping therapy, a method to correct genetic mutations in Duchenne muscular dystrophy, has been in development for many years and has shown promise.

Eligible Conditions
  • Duchenne Muscular Dystrophy

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1 year
This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
To assess the safety of CRD-TMH-001

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: Single patientExperimental Treatment1 Intervention
Single dose of CRD-TMH-001 administered by IV

Find a Location

Who is running the clinical trial?

Cure Rare Disease, IncLead Sponsor
University of Massachusetts, WorcesterOTHER
362 Previous Clinical Trials
993,045 Total Patients Enrolled
Brenda Wong, MDPrincipal InvestigatorUMass Chan Medical School
1 Previous Clinical Trials
22 Total Patients Enrolled
Medical AffairsStudy DirectorCure Rare Disease
40 Previous Clinical Trials
8,600 Total Patients Enrolled
~0 spots leftby Dec 2025
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