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 devastating diseases for which there are few or no treatment options.


To ensure that we meet our responsibility to deliver safe and effective treatments to patients, we collaborate with scientists, physicians, patients, and health authorities worldwide to conduct thorough 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 as many patients as possible suffering from these devastating diseases.

For information about our clinical trials, please visit or contact our Patient Services team at You may also have a member of your medical care team contact us at


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,” “right to try,” “compassionate use,” “early access,” or a “named patient program.” We use the term “compassionate use” to capture all of these.  

Participation in clinical trials is the most common way to access investigational therapies. Clinical trials ensure that the safety and efficacy of the investigational therapy are evaluated prior to submission for review and potential approval by regulatory authorities. Therefore, after careful consideration, Passage Bio, as a clinical-stage company, has decided that the best way for patients to access our investigational therapies is through clinical trial participation. As a result, we will not offer compassionate use at this time.

Any requests for compassionate use will be referred to study investigators to determine a patient’s eligibility for one of our ongoing clinical studies. If a patient does not qualify for one of our ongoing clinical studies, Passage Bio will direct such patients, caregivers and/or their treating physician to patient organizations and/or medical professionals who may be able to identify other clinical studies or approved treatment options. 

Once we have completed and analyzed data from our registrational trials, (i.e., a clinical trial in which the results will be used to seek regulatory approval), we will evaluate the initiation of a compassionate use program. Should changes be made to our compassionate use policy, 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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