Clinical trials · Pennsylvania
Duchenne Muscular Dystrophy clinical trials in Pennsylvania
We’re tracking 11 recruiting duchenne muscular dystrophy studies with a site in Pennsylvania — including Phase 1/Phase 2, Phase 2, Phase 2/Phase 3 and Phase 3 studies, each written for real people, not researchers.
Recruiting studies in Pennsylvania
- NCT06564974Recruiting
Long-term safety study of Agamree for Duchenne muscular dystrophy
This study watches boys with Duchenne muscular dystrophy who are already taking Agamree (vamorolone) to see how safe it is over a long period. It helps doctors understand the medicine's effects over time.
Hershey, PennsylvaniaAges 2 years+ - NCT03373968RecruitingPhase 2/Phase 3
Long-term study of givinostat safety in Duchenne muscular dystrophy
This trial looks at how safely givinostat works over a longer time in people with Duchenne muscular dystrophy (DMD). It mainly checks long-term side effects and tolerability, especially in a specific subgroup of muscle fat levels.
Philadelphia, PennsylvaniaAges 7 years+ - NCT05019625Recruiting
Study samples for biomarkers in myotonic dystrophy
This study collects blood and, for some people, muscle tissue to look for signs (biomarkers) that can help track and better understand myotonic dystrophy. You may help by providing samples and basic muscle function testing, depending on your age and condition type.
Pittsburgh, PennsylvaniaAges 5 years+ - NCT05996003RecruitingPhase 2
Exon 44 skipping drug for Duchenne muscular dystrophy
This trial tests a new drug (NS-089/NCNP-02) that aims to help boys with Duchenne muscular dystrophy produce a shorter but still working version of the dystrophin protein. It's for boys who are still walking and have a specific genetic type that can be fixed by skipping a part of the gene called exon 44.
Philadelphia, PennsylvaniaAges 4–14 - NCT06138639RecruitingPhase 1/Phase 2
Gene therapy SGT-003 for Duchenne muscular dystrophy
This trial tests a new gene therapy called SGT-003 for children with Duchenne muscular dystrophy. It aims to help the body produce a working version of dystrophin, the protein missing in Duchenne.
Philadelphia, PennsylvaniaAges birth–17 years - NCT07332013Recruiting
Urine titin test for Duchenne muscular dystrophy
This study measures a protein called titin in urine to see if it can help track muscle damage in boys with Duchenne or Becker muscular dystrophy. It also includes healthy boys for comparison.
Philadelphia, PennsylvaniaAges 2–10 - NCT01484678Recruiting
MRI and body biomarkers study for muscular dystrophy
This study uses MRI scans and blood or other biomarkers to better understand muscular dystrophy in people with Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD). It may help researchers track disease changes and identify measurable markers over time.
Philadelphia, PennsylvaniaAges 5–62 - NCT06270719Enrolling by invitation
Comparing ELEVIDYS to standard care for Duchenne muscular dystrophy
This study compares the gene therapy ELEVIDYS (delandistrogene moxeparvovec) to usual treatments for people with Duchenne muscular dystrophy (DMD). It helps researchers understand how well ELEVIDYS works over time in real-world settings.
Hershey, PennsylvaniaAges 4 years+ - NCT06925269Recruiting
DMD Voice: Interviews with patients and caregivers
This study invites people with Duchenne muscular dystrophy (DMD) and their caregivers to share their experiences through interviews. It focuses on those who have been taking the medication Givinostat for at least two years, or caregivers of patients with limited arm movement.
Yardley, PennsylvaniaAges 10 years+ - NCT06606340Enrolling by invitation
Long-term study of eteplirsen, golodirsen, or casimersen for DMD
This study follows people with Duchenne muscular dystrophy (DMD) who are taking eteplirsen, golodirsen, or casimersen. It aims to understand how these medicines work in everyday life over a long period.
Hershey, PennsylvaniaAges Any age - NCT05967351Enrolling by invitationPhase 3
Long-term follow-up for Duchenne patients treated before
This study checks how people with Duchenne muscular dystrophy are doing long-term after receiving delandistrogene moxeparvovec (SRP-9001) in an earlier trial. It helps researchers understand the long-term safety and effects of that treatment.
Philadelphia, PennsylvaniaAges Any age
Duchenne Muscular Dystrophy trials by city in Pennsylvania
Where these studies are running in Pennsylvania
Institutions with a site for the recruiting duchenne muscular dystrophy studies listed above.
- Children's Hospital of Philadelphia · 9 studies
- Penn State Milton S Hershey Medical Center · 3 studies
- University of Pittsburgh · 2 studies
- Red Nucleus
- UPMC Children's Hospital of Pittsburgh
What taking part in a duchenne muscular dystrophy study involves
A screening visit first
Before anything else, the study team checks whether you fit — usually a visit with some tests. You can stop at any point, and screening is typically free.
Care at a nearby site
Study visits happen at a clinic or hospital taking part. Many studies cover the cost of the study treatment and related visits, and some reimburse travel.
You stay in control
Taking part is voluntary and you can leave a study at any time, for any reason, without affecting your regular care.
The team decides eligibility
Our fit check is a helpful first read, not a decision. The study team makes the final call after reviewing your health history.
Duchenne Muscular Dystrophy trials in other states
Common questions
- Are there duchenne muscular dystrophy clinical trials in Pennsylvania?
- Yes. We're currently tracking 11 recruiting duchenne muscular dystrophy studies with a site in Pennsylvania, each rewritten in plain language so you can see what it's testing and who it's for.
- How do I find out if I qualify for a duchenne muscular dystrophy study in Pennsylvania?
- Each study lists its eligibility rules — age, diagnosis, prior treatments. On every trial page we explain these in plain language and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Do I have to live in Pennsylvania to take part?
- Not necessarily. These studies have a site in Pennsylvania, but eligibility is about your health, not your address — some people travel to take part, and a few studies reimburse travel. The study team can tell you what's required.
- Does it cost anything to join a duchenne muscular dystrophy trial?
- Using Clin2 is always free. Many trials cover the study treatment and related visits; some reimburse travel. The study team explains exactly what's covered before you decide.
Not the right time?
New duchenne muscular dystrophy studies open in Pennsylvania regularly. Set up a health profile and we’ll quietly watch for studies that fit you and email you when one opens.
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. Talk with your doctor about whether a specific trial is right for you.