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Orphan Diseases - Related Content

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GEN articles

• Corporate Profiles: Firm Aims to Regulate RNA in Rare Diseases
  PTC Therapeutics Focuses on Small Molecule Drugs for Genetic Disorders and Cancer

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Insight & intelligence™

• Despite Flat Budget, NIH’s TRND Program Tries to Spark Interest in Rare and Neglected Diseases
  January 31, 2012
  With 14 approved programs in total, TRND has progressed two into clinical development.
• While Advocacy Abounds, Orphan Drug Development Success Remains Elusive
  June 6, 2011
  Questions persist about whether legislature can translate ongoing research into medications.
• Changes Brewing at the FDA for Orphan Drugs
  September 13, 2010
  Initiatives to de-risk drug development could include redesigning clinical trial requirements.

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GEN news highlights

• Genzyme Gets FDA, EMA Approvals for Production Plant in Waterford, Ireland
  
• Protalix, Pfizer Report FDA Approval of Plant-Derived Gaucher Disease ERT Elelyso
  
• Synageva, Mitsubishi Tanabe Pharma Add Second Rare Disease Program to Alliance
  
• Chronix Opens South Dakota Sequencing Lab for Cancer and Rare Disease Services
  
• 4s3 Raises $20M to Create Protein Therapies for Orphan Neuromuscular Disorders
  

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GEN podcasts

• GRANDRx—Mission, Goals, and Challenges
  Interview with Patrick Terry, Acting CEO of the Grand Therapeutics Foundation, and Christopher Austin, M.D., Director of the NIH Chemical Genomics Center, and Member, Editorial Board, ASSAY and Drug Development Technologies.

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