San Diego, CANCT04174157Now EnrollingIRB Ready

Spinal Muscular Atrophy (SMA) Clinical Trial in San Diego, CA

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

Sponsored by Novartis Pharmaceuticals

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Expert Care in San Diego

Access spinal muscular atrophy (sma) specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related spinal muscular atrophy (sma) treatment provided free

Apply for This San Diego Location

Check if you qualify for this spinal muscular atrophy (sma) clinical trial in San Diego, CA

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

Why Participate?

  • No-Cost Study Care

  • Local to San Diego

    Convenient for CA 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 San Diego site if eligible
  4. 4Begin participation

About This Spinal Muscular Atrophy (SMA) Study in San Diego

Spinal muscular atrophy (SMA) is a neurogenetic disorder caused by a loss or mutation in the survival motor neuron 1 gene (SMN1) on chromosome 5q13, which leads to reduced SMN protein levels and a selective dysfunction of motor neurons. SMA is an autosomal recessive, early childhood disease with an incidence of 1:10,000 live births. SMA is the leading cause of infant mortality due to genetic diseases. The purpose of this registry is to assess the long term outcomes of patients with SMA in the context of advances in treatment options and also to characterize and assess long-term safety and effectiveness of OAV-101.

Sponsor: Novartis Pharmaceuticals

Who Can Participate

Inclusion Criteria

Patients treated with OAV-101 with a genetically confirmed diagnosis of SMA regardless of the date of diagnosis.
Appropriate consent/assent has been obtained for participation in the registry

Exclusion Criteria

\- Currently enrolled in an interventional clinical trial involving an investigational medicinal product to treat SMA. Note: Patients who are participating in a Compassionate Use Program (CUP) for OAV-101 (Zolgensma) such as a Managed Access Program (MAP), an Expanded Access Program (EAP), Single Patient Investigational New Drug (IND) (SPI) or Named Patient Program (NPP) are eligible to enroll in the registry regardless of the date of a genetic or clinical diagnosis of SMA.

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 San Diego?

Yes, this clinical trial (NCT04174157) has an active research site in San Diego, CA 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 (SMA) Treatment Options in San Diego, CA

If you're searching for spinal muscular atrophy (sma) treatment options in San Diego, CA, this clinical trial (NCT04174157) 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 San Diego research site is actively enrolling participants for this clinical trial. You'll receive care from experienced spinal muscular atrophy (sma) 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 (sma) clinical trials near you to find additional studies recruiting in your area.

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Secure · Expert Care · San Diego, CA