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    • Infantile Neuroaxonal Dystrophy (INAD)
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HomePatient Resources

Patient Resources

Clinical Trials

Before a new therapy can be made available to the public, regulated clinical trials involving patient volunteers must be conducted to evaluate the safety and effectiveness of the therapy. Our priority is to meet the needs of the patient with diligence, commitment, and compassion. Our focus on the patient perspective guides our clinical development programs along every step of the way. We strive to learn from patients, families, and caregivers and understand their daily challenges, aspirations, and perspectives. We all share a common commitment to improving the lives of everyone affected by these devastating diseases.

To find information on clinical trials that are being conducted, including those in the area of degenerative diseases, please visit www.clinicaltrials.gov. This web-based resource, which is hosted by the National Institutes of Health (NIH), is a database of privately and publicly funded clinical studies being conducted around the world.


Clinical Trials being conducted by Retrotope
(opens in a new window on www.clinicaltrials.gov):

Infantile neuroaxonal dystrophy (INAD): Friedreich’s ataxia (FA) Amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease) Progressive Supranuclear Palsy (PSP)
Phase 2/3 Study:

NCT03570931

Phase 2/3 Study:

NCT04027816

Phase 2 Study:

NCT04762589

Phase 2 Study:

NCT04937530

Natural History Study:

NCT04027816

Additional Resources

There are several organizations and patients’ advocacy groups, where you can find additional information and support. Please note that Retrotope does not guarantee the accuracy or completeness of the information contained on any third-party sites, nor does it endorse any of the opinions or information contained on those sites.

The following is a list of additional web-based resources that offer information on those conditions currently being targeted by Retrotope with its pipeline of novel drug candidates.


INAD FA ALS Progressive Supranuclear Palsy (PSP)
INADcure Foundation: https://www.inadcure.org/ Friedreich’s Ataxia Research Alliance: https://curefa.org/ The ALS Association: www.als.org CurePSP: https://www.psp.org/
National Organization for Rare Disorders (NORD): https://rarediseases.org/rare-diseases/infantile-neuroaxonal-dystrophy/ NORD: https://rarediseases.org/rare-diseases/friedreichs-ataxia/ Muscular Dystrophy Association: https://www.mda.org/ NORD: https://rarediseases.org/rare-diseases/progressive-supranuclear-palsy/
NORD: https://rarediseases.org/rare-diseases/amyotrophic-lateral-sclerosis/
Retrotope does not guarantee the accuracy or completeness of the information contained on any third-party sites, nor does it endorse any of the opinions or information contained on those sites.


Please note that when visiting any of these third-party websites, you are leaving Retrotope’s website.  Retrotope does not guarantee the accuracy or completeness of the information contained on any third-party sites, nor does it endorse any of the opinions or information contained on those sites.

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