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Ara Parseghian Medical Research Fund
United States
Приєднався 27 лип 2016
Niemann-Pick Type C (NPC) is a rare and deadly neurodegenerative disease that primarily strikes children before or during adolescence. An inherited cholesterol metabolism disorder, NPC strikes one in every 150,000 children. Notre Dame, in partnership with the Ara Parseghian Medical Research Foundation, is actively engaged in research seeking a cure for NPC.
NPC PFDD - Open Comments - Current approach to treating NPC (Late Infantile and Juvenile)
NPC PFDD - Facilitated Discussion Topic 2: Current Approach to Treating Niemann-Pick Type C (Late Infantile and Juvenile Forms)
niemannpickc-pfdd.com/
The Niemann-Pick Type C (NPC) externally led Patient-Focused Drug Development (PFDD) meeting is a groundbreaking initiative that will give persons with NPC disease and their advocates the opportunity to provide the Food and Drug Administration (FDA), drug developers, and NPC stakeholders with perspectives from our community on a number of important issues.
The meeting will follow the successful model developed by the FDA for similar Agency-convened meetings and provide persons with NPC the unique opportunity to share their story and experiences-including the challenges they face on a daily basis in living with NPC and their hopes for future treatments-with senior FDA officials and industry to better inform how therapies are developed and how the agency evaluates and considers potential therapies for the disease.
You can learn more about the NPC PFDD meeting by clicking on the link below:
niemannpickc-pfdd.com/meeting/
We would like to thank our collaborating organizations for this important effort, which include the Dana’s Angels Research Trust, Firefly Fund, Hide & Seek Foundation, Hope for Marian, Johnathan’s Dream, National Niemann-Pick Disease Foundation, and Niemann-Pick Canada.
We would also like to thank the organizations that are assisting in underwriting the cost of the PFDD meeting, which include Mallinckrodt Pharmaceuticals, Orphazyme, StrideBio, Inc., and the Together Strong NPC Foundation. These organizations will not receive any special access or benefits in regard to the meeting or related materials, and the PFDD meeting and access to the Voice of the Patient report are open to all other interested industry, foundation, and government stakeholders.
niemannpickc-pfdd.com/
The Niemann-Pick Type C (NPC) externally led Patient-Focused Drug Development (PFDD) meeting is a groundbreaking initiative that will give persons with NPC disease and their advocates the opportunity to provide the Food and Drug Administration (FDA), drug developers, and NPC stakeholders with perspectives from our community on a number of important issues.
The meeting will follow the successful model developed by the FDA for similar Agency-convened meetings and provide persons with NPC the unique opportunity to share their story and experiences-including the challenges they face on a daily basis in living with NPC and their hopes for future treatments-with senior FDA officials and industry to better inform how therapies are developed and how the agency evaluates and considers potential therapies for the disease.
You can learn more about the NPC PFDD meeting by clicking on the link below:
niemannpickc-pfdd.com/meeting/
We would like to thank our collaborating organizations for this important effort, which include the Dana’s Angels Research Trust, Firefly Fund, Hide & Seek Foundation, Hope for Marian, Johnathan’s Dream, National Niemann-Pick Disease Foundation, and Niemann-Pick Canada.
We would also like to thank the organizations that are assisting in underwriting the cost of the PFDD meeting, which include Mallinckrodt Pharmaceuticals, Orphazyme, StrideBio, Inc., and the Together Strong NPC Foundation. These organizations will not receive any special access or benefits in regard to the meeting or related materials, and the PFDD meeting and access to the Voice of the Patient report are open to all other interested industry, foundation, and government stakeholders.
Переглядів: 100
Відео
NPC PFDD - Welcome Remarks, Meeting Agenda and Desired Outcomes
Переглядів 695 років тому
NPC PFDD - Welcome Remarks Sean Kassen, PhD Director, Ara Parseghian Medical Research Fund NPC PFDD - Meeting Agenda and Desired Outcomes Timothy R. Franson, MD Moderator niemannpickc-pfdd.com/ The Niemann-Pick Type C (NPC) externally led Patient-Focused Drug Development (PFDD) meeting is a groundbreaking initiative that will give persons with NPC disease and their advocates the opportunity to ...
NPC PFDD - FDA's Approach to Rare Disease Drug Development and the Importance of the Patient Voice
Переглядів 1175 років тому
NPC PFDD - FDA’s Approach to Rare Disease Drug Development and the Importance of the Patient Voice Dragos Roman, MD Acting Director, Division of Gastroenterology and Inborn Errors Products, Food and Drug Administration niemannpickc-pfdd.com/ The Niemann-Pick Type C (NPC) externally led Patient-Focused Drug Development (PFDD) meeting is a groundbreaking initiative that will give persons with NPC...
NPC PFDD - Overview of Niemann-Pick Type C Disease and Current Treatments
Переглядів 9575 років тому
NPC PFDD - Overview of Niemann-Pick Type C Disease and Current Treatments Marc C. Patterson, MD Professor of Neurology, Pediatrics and Medical Genetics, Mayo Clinic Children’s Center niemannpickc-pfdd.com/ The Niemann-Pick Type C (NPC) externally led Patient-Focused Drug Development (PFDD) meeting is a groundbreaking initiative that will give persons with NPC disease and their advocates the opp...
NPC PFDD - Key Points and Closing Remarks
Переглядів 355 років тому
NPC PFDD - Key Points from Today’s Discussion Timothy R. Franson, MD Moderator NPC PFDD - Closing Remarks Sean Kassen, PhD Director, Ara Parseghian Medical Research Fund niemannpickc-pfdd.com/ The Niemann-Pick Type C (NPC) externally led Patient-Focused Drug Development (PFDD) meeting is a groundbreaking initiative that will give persons with NPC disease and their advocates the opportunity to p...
NPC PFDD Testimonial - Samantha Berns
Переглядів 2875 років тому
Samantha Berns - Person with NPC disease Niemann-Pick Type C Disease Patient-Focused Drug Development Meeting Panel on Discussion Topic 1: Symptoms of Niemann-Pick Type C disease (late infantile and juvenile forms) and daily impacts that matter most
NPC PFDD Testimonial - Jenna Weets
Переглядів 4245 років тому
Jenna Weets - Mother of Jeg, a child with NPC disease Niemann-Pick Type C Disease Patient-Focused Drug Development Meeting Panel on Discussion Topic 1: Symptoms of Niemann-Pick Type C disease (late infantile and juvenile forms) and daily impacts that matter most
NPC PFDD Testimonial - Phil Marella
Переглядів 1755 років тому
Phil Marella - Father of Dana and Andrew, 2 children with NPC disease Niemann-Pick Type C Disease Patient-Focused Drug Development Meeting Panel on Discussion Topic 1: Symptoms of Niemann-Pick Type C disease (late infantile and juvenile forms) and daily impacts that matter most
NPC PFDD Testimonial - Liz Heinze
Переглядів 5635 років тому
Liz Heinze - Mother of Tyler, Katie, and Faith, 3 children with NPC disease Niemann-Pick Type C Disease Patient-Focused Drug Development Meeting Panel on Discussion Topic 1: Symptoms of Niemann-Pick Type C disease (late infantile and juvenile forms) and daily impacts that matter most
NPC PFDD Testimonial - Judy DeSouza
Переглядів 1135 років тому
Judy DeSouza - Mother of Bryanna, a child with NPC disease Niemann-Pick Type C Disease Patient-Focused Drug Development Meeting Panel on Discussion Topic 1: Symptoms of Niemann-Pick Type C disease (late infantile and juvenile forms) and daily impacts that matter most
NPC PFDD Testimonial - Cindy Parseghian
Переглядів 2655 років тому
Mother of Michael, Marcia, and Christa, 3 children with NPC disease Niemann-Pick Type C Disease Patient-Focused Drug Development Meeting Panel on Discussion Topic 1: Symptoms of Niemann-Pick Type C disease (late infantile and juvenile forms) and daily impacts that matter most
NPC PFDD Testimonial - Sara McGlocklin
Переглядів 2835 років тому
Sara McGlocklin - Mother of Marian, a child with NPC disease Niemann-Pick Type C Disease Patient-Focused Drug Development Meeting Panel on Discussion Topic 2: Current approach to treating Niemann-Pick Type C (late infantile and juvenile forms)
NPC PFDD Testimonial - Alex Kray
Переглядів 3765 років тому
Alex Kray - Father of Jasper, a child with NPC disease Niemann-Pick Type C Disease Patient-Focused Drug Development Meeting Panel on Discussion Topic 2: Current approach to treating Niemann-Pick Type C (late infantile and juvenile forms)
NPC PFDD Testimonial - Sean Recke
Переглядів 885 років тому
Sean Recke, Father of Adam, a child with NPC disease Niemann-Pick Type C Disease Patient-Focused Drug Development Meeting Panel on Discussion Topic 2: Current approach to treating Niemann-Pick Type C (late infantile and juvenile forms)
NPC PFDD Testimonial - Mary Haynes
Переглядів 1055 років тому
Mary Haynes - Mother of Rebekah, a child with NPC disease Niemann-Pick Type C Disease Patient-Focused Drug Development Meeting Panel on Discussion Topic 2: Current approach to treating Niemann-Pick Type C (late infantile and juvenile forms)
NPC PFDD Testimonial - Debbie Kaflowitz
Переглядів 3095 років тому
NPC PFDD Testimonial - Debbie Kaflowitz
NPC PFDD Testimonial - Michelle Miller
Переглядів 695 років тому
NPC PFDD Testimonial - Michelle Miller
NPC PFDD - Panel 1 - Symptoms and Daily Impacts of NPC Disease (Late Infantile and Juvenile)
Переглядів 1915 років тому
NPC PFDD - Panel 1 - Symptoms and Daily Impacts of NPC Disease (Late Infantile and Juvenile)
NPC PFDD - Open Comments - Symptoms and Daily Impacts of NPC Disease (Late Infantile and Juvenile)
Переглядів 605 років тому
NPC PFDD - Open Comments - Symptoms and Daily Impacts of NPC Disease (Late Infantile and Juvenile)
NPC PFDD - Panel 2 - Current approach to treating NPC (Late Infantile and Juvenile)
Переглядів 315 років тому
NPC PFDD - Panel 2 - Current approach to treating NPC (Late Infantile and Juvenile)
NPC PFDD - Panel 3 - Symptoms and Daily Impacts of NPC Disease (Early Infantile and Adult)
Переглядів 535 років тому
NPC PFDD - Panel 3 - Symptoms and Daily Impacts of NPC Disease (Early Infantile and Adult)
NPC PFDD - Open Comments - Symptoms and Daily Impacts of NPC Disease (Early Infantile and Adult)
Переглядів 625 років тому
NPC PFDD - Open Comments - Symptoms and Daily Impacts of NPC Disease (Early Infantile and Adult)
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