Patient and Community Resources

Support for patients and caregivers through advocacy organizations and downloadable resources

Advocacy Organizations

GM1 gangliosidosis

Cure GM1 Foundation, a nonprofit patient advocacy group, provides families caring for a child with GM1 gangliosidosis with support and resources, opportunities to meet others families who understand the challenges of GM1 gangliosidosis, and direct funding toward research for a cure.

Visit for more information and support.

National Tay-Sachs & Allied Diseases Association (NTSAD), a nonprofit patient advocacy group, is leading the worldwide fight to treat and cure Tay-Sachs, Canavan, GM1 gangliosidosis, and Sandhoff diseases by driving and funding research, forging collaboration, fostering community, and supporting families.

Visit for more information and support.

Krabbe disease

Hunter’s Hope, a nonprofit organization, was founded to provide hope to families affected by Krabbe disease and leukodystrophy through education, awareness, research, and family care. Hunter’s Hope is committed to newborn screening for every child, every time, everywhere.

Visit Hunter’s Hope for more information and support.

KrabbeConnect, a nonprofit organization, was established to address the need for information, research, support, and encouragement for patients and their families. KrabbeConnect is working toward the day when every patient affected by this disorder can receive an early diagnosis and access to state-of-the-art care so that they may live a life free of Krabbe disease.

Visit KrabbeConnect for more information and support.

Krabbe UK, a charitable health organization, provides practical support to families and children affected by Krabbe leukodystrophy. They also champion research and raise awareness of the disease.

Visit for more information and support.

United Leukodystrophy Foundation (ULF), a nonprofit voluntary health organization, is dedicated to funding cutting-edge research and to providing patients and their families with disease information and medical referrals.

Visit for more information and support.

Frontotemporal dementia

Alzheimer’s Association, a nonprofit patient advocacy organization, is leading the way to end Alzheimer’s and all other forms of dementia—by accelerating global research, driving risk reduction and early detection, and maximizing quality care and support.

Visit for more information and support.

The Association for Frontotemporal Degeneration (AFTD), a patient advocacy organization, is on a mission to improve the quality of life of people affected by frontotemporal dementia and drive research to find a cure. Every day, they work to advance research, awareness, support, education, and advocacy.

Visit for more information and support.

CurePSP, a nonprofit patient advocacy organization, is the leading source of information and support for patients and their families, other caregivers, and professionals regarding prime-of-life neurodegeneration diseases.

Visit for more information and support.

Dementia Society of America, the nation’s leading volunteer-driven all-Dementias awareness organization, the Dementia Society of America brings much-needed education, local resources, and life-enrichment to individuals and families impacted by Dementia.

Visit for more information and support.

FTD Disorders Registry, an online database, collects information from those affected by all types of frontotemporal degeneration, including FTD.

Visit for more information and support.

Living with FTD, part of the University of Pennsylvania, brings together an interdisciplinary team of clinicians and researchers dedicated to delivering the highest quality care and support to patients and caregivers affected by frontotemporal dementia and other neurodegenerative diseases.

Visit for more information and support.

World FTD United, an international group of organizations and health professionals, provides support for caregivers and all those affected by FTD.

Visit for more information and support.

Compassionate use statement

Passage Bio is dedicated to developing transformative gene therapies for patients with severe, life threatening neurologic disorders. We approach our work every day with great urgency and commitment to the patients and families who suffer from these catastrophic diseases for which there are few or no treatment options.

Our commitment to clinical research

To ensure that we meet our responsibility to deliver safe and effective treatments to patients, we are collaborating with scientists, physicians, patients and health authorities around the world to conduct rigorous clinical trials. We believe that it is through clinical trials and regulatory approval that we can ultimately ensure safe and timely access to our medicines for all patients and families suffering from these devastating diseases.

Review process for Expanded Access requests

We understand that there may be circumstances under which patients or caregivers may request treatment with our investigative therapies before they have been approved by regulatory agencies. This is sometimes called “expanded access,” or “compassionate use” or a “named patient program,” and we use the term Expanded Access to include all of these. While we do not anticipate providing for Expanded Access to investigational therapies prior to completion and analysis of our registrational clinical trials, we have developed the following process to evaluate such requests for Expanded Access:

  • Before one of our investigative therapies is approved by regulatory authorities, any requests for Expanded Access to such investigative therapy received by Passage Bio will be referred to study investigators to determine a patient’s eligibility for one of our ongoing clinical studies with that product candidate

  • If a patient does not qualify for one of our ongoing clinical studies, Passage Bio will direct such requests to patient organizations, medical professionals and/or scientists who may be able to identify other clinical studies or approved treatment options

  • Once we have completed and analyzed data from our registrational trials, we will consider requests for Expanded Access on a case-by-case basis. Requests for Expanded Access will be considered based on a number of factors, including the following factors:

    • Based on available scientific data, there is a reasonable expectation that the mechanism of action and/or biological activity of the investigational therapy will provide therapeutic benefit to the individual patient’s disease

    • Available safety and efficacy data for the investigational therapy and the potential benefits and risks for the individual patient;

    • The availability of alternative treatment options

    • Compliance with legal and health authority requirements, including institutional review board (IRB) approvals

    • Potential compromise of related clinical trials or regulatory pathways

    • Feasibility considerations including but not limited to the availability of the investigational therapy supply, and the availability of expert clinical oversight to ensure the safe administration of investigational therapy and adequate clinical follow up

Making a Request for Expanded Access

We are committed to evaluating all requests for Expanded Access in a fair and equitable manner. Requests for access to a Passage Bio investigational therapy must be made voluntarily by the patient’s treating physician. Physicians seeking Expanded Access for patients with no alternative treatment options should submit their requests to

Passage Bio will acknowledge requests from licensed physicians, generally within 10 business days of receipt and a decision will be made and communicated thereafter. Passage Bio retains full discretion to determine whether to grant requests for Expanded Access, and requests for access may not be granted, even when eligibility criteria are met.

Expanded Access to investigational therapies must comply with the applicable country-specific laws and regulations, including importation requirements, approvals from applicable regulatory bodies, and by an appropriate Institutional Review Board or Ethics Committee. Passage Bio may require agreements and/or certifications executed by the treating physician and other information to be provided prior to final approval of any request.

Patients and caregivers seeking information about access to our investigational therapies, either through a clinical trial or through Expanded Access, should consult with their treating physician.

Should changes be made to our Expanded Access programs for our investigational therapies, we will update this website.

Passage Bio may revise this Policy at any time as provided by the 21st Century Cures Act.

Genetic counseling and testing

We are collaborating with Invitae to facilitate genetic testing to support early diagnosis and greater awareness of clinical trials for patients with GM1 gangliosidosis and Krabbe disease. Invitae’s no-charge testing combined with counseling information on clinical trials enables clinicians to intervene sooner and more efficiently, shortening the timeline to reliable diagnosis. This partnership offers a key resource for patients and their families.

Our collaboration with Informed DNA involves designing and supporting a genetic screening and counseling program that will be free of charge for patients with FTD.



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