Externally-Led Patient-Focused Drug D... - Leukodystrophy Su...

Leukodystrophy Support

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Externally-Led Patient-Focused Drug Development meeting (EL-PFDD)

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We are thrilled to announce that we will be hosting an Externally-Led Patient-Focused Drug Development meeting (EL-PFDD) on July 22, 2022.

Our proposed target patient population for this meeting is Adrenoleukodystrophy (ALD) in Adulthood:

Men with adrenomyeloneuropathy (AMN)

Men with cerebral ALD

Symptomatic women with ALD

The meeting will be held virtually, which will allow increased participation across the United States and the world. The meeting will be centered around patient and caregiver perspectives, which are critical to help provide context when FDA makes regulatory decisions for new drugs. We will ask community members to participate by calling in, answering poll questions, and writing comments.

We believe this EL-PFDD meeting will allow the FDA and other key stakeholders to obtain a wide range of patient, caregiver, and healthcare provider input on ALD. This includes their perspectives on their condition, its impacts on daily life, the landscape of current treatment options, as well as input into outcomes and patient preferences for future therapies.

Our goal is to publish a Voice of the Patient Report that documents the severe disease burden and unmet medical need of ALD and AMN in patients' own voices. Our hope is that it will influence every stage of drug development.

Please save the date and watch for details in the coming months.