Research Programs & Resources

Audentes Research Programs & Resources

Audentes is currently conducting investigational research in the following different rare conditions. As part of our commitment to the patients and families we work with, we are constantly gathering information and resources related to these specific areas. We hope that you find some helpful information here.

Please click below for information and resources related to a specific condition.

Below are patient advocacy organizations that are not specific to any one rare condition, but instead serve the broader needs of those affected by a rare condition. For patient advocacy organizations related to a certain condition, please visit the specific program page.

ARM Foundation

The Alliance for Regenerative Medicine (ARM) Foundation for Cell and Gene Medicine is dedicated to providing the education, information and research needed to accelerate patient awareness of and access to transformative therapies.

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Caregiver Action Network

Caregiver Action Network (CAN) is a family caregiver organization working to improve the quality of life for the more than 90 million Americans who care for loved ones with chronic conditions, disabilities, disease, or the frailties of old age. CAN is a non-profit organization providing education, peer support, and resources to family caregivers across the country free of charge.

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EURORDIS – Rare Diseases Europe is a non-governmental patient-driven alliance of rare disease patient organizations representing 804 rare disease patient organizations in 69 countries.

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EveryLife Foundation

The EveryLife Foundation for Rare Diseases is dedicated to accelerating biotech innovation for rare disease treatments through science-driven public policy.

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Global Genes

Global Genes is a rare disease patient advocacy organization that builds awareness, educates the global community, and provides critical connections and resources that equip advocates to become activists for their disease.

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Muscular Dystrophy Association (MDA)

The Muscular Dystrophy Association’s mission is to foster and promote the alleviation of the conditions of persons with muscular dystrophy and related neuromuscular disorders through support of basic and applied research seeking the causes of and effective treatments for these diseases.

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Needy Meds

NeedyMeds is a 501(c)(3) national non-profit information resource dedicated to helping people locate assistance programs to help them afford their medications and other healthcare costs.

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National Organization for Rare Disorders (NORD) is a patient advocacy organization dedicated to improving the lives of individuals and families living with rare diseases and the organizations that serve them. NORD, along with its more than 280 patient organization members, is committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and patient services.

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Rare Advocacy Movement (RAM) is a patient advocacy initiative focused on documenting the complex structure and unique dynamics of the rare disease patient advocacy landscape.

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Rare Revolution

Rare Revolution is a digital magazine giving a voice to patients affected by rare conditions and the charities that represent and support them.

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What other kinds of support do you seek? Whether it be finding a canine companion, support for siblings, or clothing and equipment specifically designed for a rare condition, we would love to list other organizations that support those with rare conditions in other ways.

Thoughts or recommendations? Send us an email at

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