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Rare Diseases and Orphan Products Accelerating Research and Development (2010) / Chapter Skim
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References
Pages 249-282

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From page 249...
... 2010. Clinical Trials: State Laws Regarding Insurance Coverage.
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From page 250...
... 2009. Statement Presented by Susan Alpert to IOM Committee on Accelerating Rare Diseases Research and Orphan Product Development, Washington, DC.
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From page 251...
... Presentation to the IOM Committee on Accelerating Rare Diseases Research and Orphan Product Development, Washington, DC. February 4.
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From page 252...
... OJRD (Orphanet Journal of Rare Diseases)
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From page 253...
... 2009. Lentiviral-mediated gene therapy of hematopoietic stem cells delays disease progression in patients with a fatal brain disorder.
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From page 254...
... 2010a. Guidance for Industry: Adaptive Design Clinical Trials for Drugs and Biologics (Draft)
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From page 255...
... http://www.fda.gov/downloads/Medical Devices/DeviceRegulationandGuidance/GuidanceDocuments/ucm071121.pdf (accessed August 24, 2010)
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From page 256...
... Presentation to IOM Committee on Ac celerating Rare Diseases Research and Orphan Product Development, Washington, DC. August 12.
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From page 257...
... 2009. Big PhRMA adopting orphan drug strategy.
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From page 258...
... 2007. Testimony submitted by the National Organization for Rare Disorders before the Social Security Administration Public Hearing on Compassionate Allowances, Washington, DC.
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From page 259...
... 2009. Accelerating rare diseases research and orphan product development: a FARA [Friedreich's Ataxia Research Alliance]
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From page 260...
... 2009c. Orphan Products: Hope for People with Rare Diseases.
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From page 261...
... Presentation (by phone) to the IOM Committee on Accelerating Rare Diseases Research and Orphan Product Development, Washington, DC.
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From page 262...
... 2008. The Orphan Drug Act turns 25.
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From page 263...
... 2006. Adopting orphan drugs -- two dozen years of treating rare diseases.
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From page 264...
... process. Presentation to the IOM Committee on Accelerating Rare Diseases Research and Orphan Product Development, Washington, DC.
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From page 265...
... 2006. Safe Medical Devices for Children, edited by M
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From page 266...
... Pre sentation on behalf of the Multiple Myeloma Research Foundation and the Multiple Myeloma Research Consortium to the IOM Committee on Accelerating Rare Diseases Research and Orphan Product Development, Washington, DC. November 23.
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From page 267...
... 2010. Strategy reveals rare disease genes.
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From page 268...
... Presentation to IOM Committee on Accelerating Rare Diseases Research and Orphan Product Development, Washington, DC. February 4.
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From page 269...
... 2010c. Push to cure rare diseases.
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From page 270...
... 2002. Orphan products -- pain relief for clinical development headaches.
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From page 271...
... Nature Reviews Drug Discovery 8(12)
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From page 272...
... 2002. Report on Steps to Coordinate Rare Diseases Research Programs.
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From page 273...
... Rare Diseases and Related Terms. http://rarediseases.info.nih.gov/RareDisease List.aspx (accessed August 4, 2010)
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From page 274...
... Presentation to IOM Committee on Accelerating Rare Diseases Research and Orphan Product Development, Washington, DC. February 4.
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From page 275...
... . Statement to IOM Committee on Accelerating Rare Diseases Research and Orphan Product Development, Washington, DC.
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From page 276...
... 2010. Creating a global rare disease patient registry linked to a rare diseases biorepository database: Rare Disease-HUB (RD-HUB)
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From page 277...
... Orphanet Journal of Rare Diseases 3(33)
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From page 278...
... http://www.spiration.com/downloads/NTAPPress ReleaseFinal.pdf (accessed August 3, 2010)
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From page 279...
... Presentation to IOM Committee on Accelerating Rare Diseases Research and Orphan Product Develop ment, Washington, DC. February 4.
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From page 280...
... Presentation to IOM Committee on Ac celerating Rare Diseases Research and Orphan Product Development, Washington, DC. February 4.
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From page 281...
... 2009. Novartis shifts focus to rare diseases.
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From page 282...
... eMedicine April 16. http://emedicine.medscape.com/article/956936-treatment (accessed August 11, 2010)
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Key Terms

  • rare diseases
  • orphan drug
  • orphan products
  • accessed september
  • diseases research

  • orphan product
  • accelerating rare
  • gene therapy
  • drug discovery
  • medical devices

  • clinical trials
  • newborn screening
  • cessed august
  • rare disease
  • health affairs

  • approval letter
  • york times
  • drug law
  • drug act
  • press release

  • street journal
  • orphan diseases
  • drug development
  • rare disorders
  • clinical pharmacology

  • american journal
  • fda regulation
  • critical path
  • orphan drugs
  • drug evaluation


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