fda marks orphan drug act milestone: 30 year recognition january 2013
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FDA Marks Orphan Drug Act Milestone: 30 Year Recognition January 2013. One of Many Rare Disease Heroes…. Henry Waxman and Orrin Hatch Author and cosponsors of the Orphan Drug Act (ODA). One of Many Rare Disease Heroes…. Abbey Meyers - PowerPoint PPT PresentationTRANSCRIPT
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FDA Marks Orphan Drug Act Milestone:
30 Year RecognitionJanuary 2013
![Page 2: FDA Marks Orphan Drug Act Milestone: 30 Year Recognition January 2013](https://reader035.vdocument.in/reader035/viewer/2022070420/56815da4550346895dcbd1a1/html5/thumbnails/2.jpg)
One of Many Rare Disease Heroes…
Henry Waxman and Orrin Hatch
Author and cosponsors of the Orphan Drug Act (ODA)
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One of Many Rare Disease Heroes…
Abbey MeyersChampion for ODA and founder of
National Organization for Rare Disorders
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One of Many Rare Disease Heroes…
Media/Entertainment
IndustryRaising awareness about rare diseases
and the need for medical products
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One of Many Rare Disease Heroes…
Rare Disease Advocacy Groups
Organization, education, support, research, advocacy, and action
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One of Many Rare Disease Heroes…
FDA Orphan Incentive Programs
Advancing orphan products through orphan designations, grants, and
facilitation
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One of Many Rare Disease Heroes…
NIH Office of Rare Diseases
ResearchAdvancing rare disease information,
collaboration, and research
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One of Many Rare Disease Heroes…
Robert Campbell Jr, MD
representing Clinician/product developers and advocates;Developed the artificial rib humanitarian use
device
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One of Many Rare Disease Heroes…
Hemin and Desiree Lyon
representing1st approved orphan designated drug
and patient advocacy
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One of Many Rare Disease Heroes…
Jana Monacorepresenting
Parent advocacy for newborn screening and medical foods
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One of Many Rare Disease Heroes…
Industry-Investors-Academia
Developing products in the spirit of the ODA
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One of Many Rare Disease Heroes…
Sharon Terryrepresenting
Advocacy for innovative and collaborative approaches for rare disease products
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One of Many Rare Disease Heroes…
NIH Undiagnosed Diseases Program
Cross-cutting efforts to understand and treat rare diseases
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One of Many Rare Disease Heroes…
Nancy Wexler, PhD
representingPatient advocacy and gene
discovery/research
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One of Many Rare Disease Heroes…
Ashley Appellrepresenting
Patients living and thriving with a rare disease
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One of Many Rare Disease Heroes…
FDA Offices/Centers
Communication, diagnostic and therapeutic product evaluation, safety, and access
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One of Many Rare Disease Heroes…
Myozyme and Tiffany
Houserepresenting
Contributions of patient representatives to the FDA orphan approval process
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One of Many Rare Disease Heroes…
Ronald J. Bartekrepresenting
Family member founder of a rare disease advocacy group
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One of Many Rare Disease Heroes…
Pediatric Ventricular Assist Device-Berlin
Heartrepresenting
ODA Grants and Humanitarian Use Devices for pediatric rare diseases
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One of Many Rare Disease Heroes…
Denise Ney, PhDrepresenting
Clinical dietitian/medical foods researcher for metabolic disorders
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One of Many Rare Disease Heroes…
Hal Dietz, MDrepresenting
Clinical research in rare diseases
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One of Many Rare Disease Heroes…
FDA/CDER Rare Diseases Program
Focused regulatory science and education efforts for rare disease drug
development
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One of Many Rare Disease Heroes…
NIH TRND Program
Stimulating drug discovery and collaborative preclinical development for rare disease
drugs
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One of Many Rare Disease Heroes…
EMA/EURORDIS/IRDIRC/ICORD
representingCollaborative international rare disease
efforts
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One of Many Rare Disease Heroes…
Pearl O’Rourke, MD
representingIRB efforts on behalf of rare diseases
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One of Many Rare Disease Heroes…
Jouni Uitto, MD, PhD
representingBasic research in rare diseases
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One of Many Rare Disease Heroes…
George Harris Carterrepresenting
Patient advocacy for drug development and clinical trials
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One of Many Rare Disease Heroes…
Kalydecorepresenting
Personalized medicines for rare diseases
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One of Many Rare Disease Heroes…
Leslie Boyer, MDrepresenting
Academic grantees successfully pursuing biological marketing approvals
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One of Many Rare Disease Heroes…
Rodney Howell, MD
representingAdvocacy for newborn screening/early detection and treatment of congenital
disorders
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FDA Marks Orphan Drug Act Milestone:
30 Year RecognitionJanuary 2013