Atlanta, GANCT05861986Now EnrollingIRB Ready

Spinal Muscular Atrophy Clinical Trial in Atlanta, GA

Access cutting-edge spinal muscular atrophy treatment through this clinical trial at a research site in Atlanta. Study-provided care at no cost to qualified participants.

Sponsored by Hoffmann-La Roche

Quick Self-Assessment

See if you qualify for this Atlanta location

Preparing your pre-screening questions...

Expert Care in Atlanta

Access spinal muscular atrophy specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related spinal muscular atrophy treatment provided free

Apply for This Atlanta Location

Check if you qualify for this spinal muscular atrophy clinical trial in Atlanta, GA

Secure & Confidential

Your information is protected and will only be shared with the research team.

Why Participate?

  • No-Cost Study Care

  • Local to Atlanta

    Convenient for GA residents

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3Visit Atlanta site if eligible
  4. 4Begin participation

About This Spinal Muscular Atrophy Study in Atlanta

This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered as an early intervention in pediatric participants with spinal muscular atrophy (SMA) and 2 SMN2 copies who have previously received onasemnogene abeparvovec. Participants are children \< 2 years of age genetically diagnosed with SMA.

Sponsor: Hoffmann-La Roche

Who Can Participate

Inclusion Criteria

\<2 years of age at the time of informed consent
Confirmed diagnosis of 5q-autosomal recessive SMA, including genetic confirmation of homozygous deletion or compound heterozygosity predictive of loss of function of the Survival of Motor Neuron 1 (SMN1) gene
Confirmed presence of two SMN2 gene copies as documented through laboratory testing
Administration of onasemnogene abeparvovec pre-symptomatically or post-symptomatically
Has received onasemnogene abeparvovec for SMA no less than 13 weeks, but not more than months 30 weeks, prior to enrollment
If treated with risdiplam prior to onasemnogene abeparvovec, risdiplam treatment must not have exceeded 3 weeks and must be discontinued 1 day prior to onasemnogene abeparvovec administration
Has, in the opinion of the investigator, not experienced clinically significant decline in function from the time of onasemnogene abeparvovec administration

Exclusion Criteria

Previous or current enrolment in invest

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in Atlanta?

Yes, this clinical trial (NCT05861986) has an active research site in Atlanta, GA that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

Spinal Muscular Atrophy Treatment Options in Atlanta, GA

If you're searching for spinal muscular atrophy treatment options in Atlanta, GA, this clinical trial (NCT05861986) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our Atlanta research site is actively enrolling participants for this clinical trial. You'll receive care from experienced spinal muscular atrophy specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all spinal muscular atrophy clinical trials near you to find additional studies recruiting in your area.

More Pulmonary Hypertension Trials in Atlanta, GA

See all pulmonary hypertension clinical trials recruiting in Atlanta — not just this study.

Browse Pulmonary Hypertension Trials in Atlanta

Ready to Join in Atlanta?

Take the first step toward participating in this groundbreaking clinical trial

Secure · Expert Care · Atlanta, GA