voice of the patient report

The "Dravet Syndrome Voice of the Patient" report is a comprehensive document created from the feedback gathered at the Dravet syndrome EL-PFDD meeting. It includes shared testimony and data from attendees who participated via webcast, as well as submitted comments before and after the meeting.

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When was the Alport Syndrome meeting?

On August 3, 2018 , the National Kidney Foundation (NKF) and Alport Syndrome Foundation (ASF) held . an Externally Led Patient-Focused Drug Development (EL-PFDD) Meeting on Alport Syndrome (AS). The . broad goals of the meeting were to inform the Food and Drug Administration (FDA) and other .

What is the 569C report?

Pursuant to section 569C of the Federal Food, Drug, and Cosmetic Act to: . Center for Drug Evaluation and Research (CDER) . U.S. Food and Drug Administration (FDA) . This report reflects the National Kidney Foundations and Alport Syndrome Foundations accounts of the .

What is Alport syndrome?

Alport syndrome is a rare, inherited disorder of type IV collagen, caused by mutations in the COL4A3, . COL4A4, andCOL4A5 genes. Under physiological conditions, the collagen subunits produced by each of . these genes (α-3, α-4, and α-5, respectively) come together to form a triple helix, the building block for .

When will the FSHD Society be a virtual voice of the patient?

In June 2020, the FSHD Society convened a virtual Voice of the Patient Forum to enable the U.S. Food and Drug Administration (FDA) and other important drug development stakeholders to hear directly from patients and caregivers about their journey with FSHD.

Why did the woman die without a tracheostomy?

She went to the hospital and their pulmonologist told us that due to her blood pressure dropping so low when she coughed and her weakness from FSHD, she would die without a tracheostomy, which they performed against her wishes. What should have been a short-term solution, quickly became permanent.

When was the FDA meeting on hereditary angioedema?

On September 25, 2017 , FDA is conducting a public meeting on Patient-Focused Drug Development for Hereditary Angioedema. FDA is interested in obtaining patient and caregiver perspectives on the on the impact of Hereditary Angioedema on daily life and patient views on treatment approaches.

When was the FDA meeting for systemic sclerosis?

On October 13, 2020 , FDA hosted a public meeting on Patient-Focused Drug Development for Systemic Sclerosis. FDA was interested in hearing perspectives from individuals with systemic sclerosis on the health effects and daily impacts of their systemic sclerosis, treatment goals, and decision factors considered when seeking out or selecting a treatment.

When was the FDA meeting on Chagas disease?

On April 28, 2015, FDA conducted a public meeting on Patient-Focused Drug Development for Chagas Disease. FDA was interested in obtaining patient perspectives on the impact of Chagas Disease on daily life and patient views on treatment approaches.