Clinical trials · Philadelphia, PA
Spinal Muscular Atrophy clinical trials in Philadelphia
We’re tracking 7 recruiting spinal muscular atrophy studies with a site in or near Philadelphia — including Phase 1, Phase 2 and Phase 4 studies, each written for real people, not researchers.
Recruiting studies near Philadelphia
- NCT05866419Recruiting
Study of a port and catheter system for spinal muscular atrophy
This study tests a new device called the ThecaFlex DRx System, which is a small port and catheter placed under the skin to deliver Spinraza directly into spinal fluid—instead of needing repeated spinal taps. The device aims to make receiving medication easier and safer for people with SMA.
Philadelphia, PennsylvaniaAges 3 years+ - NCT07287982RecruitingPhase 2
ARGX-119 for children with SMA who can walk
This study tests a new medicine called ARGX-119 in children with SMA. It is given through an IV and aims to help kids who can already walk get stronger and stay healthy.
Philadelphia, PennsylvaniaAges 5–17 - NCT05861986RecruitingPhase 4
Risdiplam for very young children after gene therapy for SMA
This trial studies whether risdiplam, given early after gene therapy, is safe and helps children with spinal muscular atrophy (SMA) keep improving or stay stable. It focuses on children under 2 years old who already received gene therapy and need to meet timing and health requirements.
Philadelphia, PennsylvaniaAges 3 months–2 years - NCT05966038Recruiting
Study of ALS and related movement nerve conditions
This study collects and stores medical information from people diagnosed with ALS or certain related conditions affecting movement. It may help researchers better understand how these illnesses change over time and improve future treatments.
Philadelphia, PennsylvaniaAges 18–90 - NCT05861999RecruitingPhase 4
Risdiplam for young children with SMA after gene therapy plateau
This trial studies whether risdiplam is safe and helpful for children under 2 with spinal muscular atrophy (SMA) whose function has leveled off or declined after gene therapy. It may be an option if you saw benefits from gene therapy but then noticed a stop or setback in key abilities.
Philadelphia, PennsylvaniaAges 3 months–2 years - NCT06555419RecruitingPhase 1
How Nusinersen works with ThecaFlex DRx system
This study looks at how a medicine called Nusinersen (Spinraza) works in the body when given through a new device called ThecaFlex DRx. It is for people with spinal muscular atrophy who are already on a steady dose of Nusinersen and are part of the main PIERRE study.
Philadelphia, PennsylvaniaAges 3 years+ - NCT07047144RecruitingPhase 2
Apitegromab for infants with SMA under 2 years old
This study tests a new medicine called apitegromab for babies under 2 years old with spinal muscular atrophy (SMA). It looks at whether the drug can help with muscle strength and reaching motor milestones like sitting or rolling.
Philadelphia, PennsylvaniaAges Up to 2 years
Hear when a new Spinal Muscular Atrophy trial opens near Philadelphia, PA
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Spinal Muscular Atrophy trials in other cities
Where these studies are running near Philadelphia
Institutions with a site for the recruiting spinal muscular atrophy studies listed above.
- Children's Hospital of Philadelphia · 6 studies
- Temple University Hospital
- Thomas Jefferson University Hospital
What taking part in a spinal muscular atrophy 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.
Looking wider? See all recruiting spinal muscular atrophy studies in Pennsylvania.
Common questions
- Are there spinal muscular atrophy clinical trials near Philadelphia?
- Yes. We're currently tracking 7 recruiting spinal muscular atrophy studies with a site near Philadelphia, 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 spinal muscular atrophy study near Philadelphia?
- 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 Philadelphia to take part?
- Not necessarily. These studies have a site near Philadelphia, 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 spinal muscular atrophy 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 spinal muscular atrophy studies open near Philadelphia regularly. Set up a health profile and we’ll quietly watch for studies that fit you and email you when one opens.