Skip to main content

FDA Approves PTC’s Gene Therapy for AADC Deficiency

Patient Resources

Welcome to Insightful Moments™

Insightful Moments is a community education series of programs and materials created to support patients with rare disease and their families.

Insightful Moments My VIBE™

Listen to patients and their families as they share their unique experiences living with a rare disease – from diagnosis to overcoming challenges and building a supportive community.

Click here to listen to the podcast
MyVIBE Logo

Insightful Moments Educational Series Materials

Choose your educational series and view PDFs, videos, and webinar recordings to help you along your journey.

Below, you will find pdf roadmaps to help you along your journey. Click the tile to open a single pdf, or check the box to download multiple pdfs at one time. Videos will open in a video player, so you can view them directly in your browser. You can navigate between the Education Series’ by clicking the category button.

We are here to help! Email us at InsightfulMoments@ptcbio.com, or call 1-833-PTC-HOPE, 1-833-PTC-VIDA (Spanish)

Para obtener más información sobre Momentos de Claridad, por favor visite https://ptcmomentosdeclaridad.com o comuníquese con Enlace de Pacientes.

All Educational Series Materials

  1. Insightful Moments - Accessible College - Preparing a Middle Schooler for College
    Preparing a middle schooler for college
    Accessible College
  2. Insightful Moments - Video - Accessible College - Preparing a Middle Schooler for College
    Preparing a middle schooler for college (webinar recording)
    Accessible College
  3. Insightful Moments - Accessible College - Developing Independent Living and Executive Function Skills
    Developing independent living skills
    Accessible College
  4. Insightful Moments - Video - Accessible College - Developing Independent Living and Executive Function Skills
    Developing independent living skills (webinar recording)
    Accessible College
  5. New
    Insightful Moments - Accessible College - Transition to College with a Physical Disability or Chronic Health Condition
    Transition to college with a physical disability or chronic health condition
    Accessible College
  6. New
    Insightful Moments Video
    Transition to college with a physical disability (webinar recording)
    Accessible College
  7. New
    Insightful Moments - Accessible College - Going to College with a PCA
    Going to college with a personal care attendant
    Accessible College
  8. New
    Insightful Moments - Accessible College - Going to college with a Personal Care Attendant
    Going to college with a personal care attendant (webinar recording)
    Accessible College
  9. Insightful Moments - Sibling Support - Parent Roadmap
    Parent roadmap
    Sibling Support
  10. Insightful Moments - Sibling Support - Video - The Impact of Rare Disease on Siblings
    The impact of rare disease on siblings: A parent perspective (webinar recording)
    Sibling Support
  11. Insightful Moments - Sibling Support - Sibling Roadmap
    Siblings roadmap
    Sibling Support
  12. Insightful Moments - Sibling Support - Video - Supporting Rare Siblings
    Sibling support (webinar recording)
    Sibling Support
  13. New
    Understanding your prescription
    Drug Discovery and Development
  14. Insightful Moments - Drug Discovery and Development - Understanding the FDA Approval Process for Gene Therapies
    Understanding the FDA approval process for gene therapies
    Drug Discovery and Development
  15. Insightful Moments - Drug Discovery and Development - Discovery Development and Regulatory
    Discovery, development & regulatory
    Drug Discovery and Development
  16. Insightful Moments Video
    Drug discovery (video)
    Drug Discovery and Development
  17. Insightful Moments - Drug Discovery and Development - Manufacturing and Commercialization
    Manufacturing & commercialization
    Drug Discovery and Development
  18. Insightful Moments Video
    Drug manufacturing (video)
    Drug Discovery and Development
  19. Insightful Moments Video
    Drug commercialization (video)
    Drug Discovery and Development
  20. Insightful Moments Video
    What are clinical trials (video)
    Navigating Clinical Trials
  21. Insightful Moments - Navigating Clinical Trials - Finding a Clinical Trial
    Finding a clinical trial
    Navigating Clinical Trials
  22. Insightful Moments Video
    Finding a clinical trial (video)
    Navigating Clinical Trials
  23. Insightful Moments - Navigating Clinical Trials - Participant Journey
    Participant journey
    Navigating Clinical Trials
  24. Insightful Moments - Navigating Clinical Trials - Questions to Consider
    Questions to consider
    Navigating Clinical Trials
  25. Insightful Moments Video
    Questions to consider (video)
    Navigating Clinical Trials
  26. Insightful Moments - Navigating Clinical Trials - Understanding the Informed Consent Form
    Understanding the informed consent form
    Navigating Clinical Trials
  27. Insightful Moments - Navigating Clinical Trials - Natural History Studies
    Natural history studies
    Navigating Clinical Trials
  28. Insightful Moments - Discovering Wellness - Handling Emotions in the Clinical Trial Decision Process
    Handling emotions in the clinical trial decision process
    Discovering Wellness
  29. Insightful Moments Video
    Handling emotions in the clinical trial decision process
    Discovering Wellness
  30. Insightful Moments - Discovering Wellness - Coping and Accepting the Rare Disease Diagnostic Journey
    Coping and accepting the rare disease diagnostic journey
    Discovering Wellness
  31. Insightful Moments Video
    Coping and accepting the rare disease diagnostic journey
    Discovering Wellness
  32. Insightful Moments - Discovering Wellness - Navigating Life with a Rare Disease
    Navigating life with a rare disease
    Discovering Wellness
  33. Insightful Moments Video
    Navigating life with a rare disease
    Discovering Wellness
  34. Insightful Moments Video
    Life on a waterbed: Trauma in the family system (webinar recording)
    Discovering Wellness
  35. Insightful Moments - Advocacy Works - Advocacy Works
    Advocacy works!
    Advocacy Works!
  36. Insightful Moments Video
    Advocacy Works! (webinar recording)
    Advocacy Works!
  37. Insightful Moments - Holistic Inspirations Webinar Recording
    Holistic inspirations (webinar recording)
    Holistic Inspirations
  38. Insightful Moments - Holistic Impressions - Personal Space
    Personal space
    Holistic Inspirations
  39. Insightful Moments Video
    Personal space (video)
    Holistic Inspirations
  40. Insightful Moments - Holistic Impressions - Personal Connection
    Personal connection
    Holistic Inspirations
  41. Insightful Moments Video
    Personal connection (video)
    Holistic Inspirations
  42. Insightful Moments - Holistic Impressions - Personal Preference
    Personal preference
    Holistic Inspirations
  43. Insightful Moments Video
    Personal preference (video)
    Holistic Inspirations
  44. Insightful Moments - Navigating Insurance - Insurance Terminology
    Insurance Terminology
    Navigating Insurance
  45. New
    Insightful Moments Video
    Insurance Terminology (video)
    Navigating Insurance
  46. Insightful Moments - Navigating Insurance - Different Coverages
    Different Coverages
    Navigating Insurance
  47. New
    Insightful Moments Video
    Different Coverages (video)
    Navigating Insurance
  48. Insightful Moments - Navigating Insurance - Denials and Approvals
    Denials and Appeals
    Navigating Insurance
  49. New
    Insightful Moments Video
    Denials and Appeals (video)
    Navigating Insurance
  50. Insightful Moments - Medicaid and Government Programs - Medicare and the Affordable Care Act
    Medicare and the Affordable Care Act
    Medicaid and Government Programs
  51. New
    Insightful Moments Video
    Medicare and the Affordable Care Act (video)
    Medicaid and Government Programs
  52. Insightful Moments - Medicaid and Government Programs - Medicaid and Waivers
    Medicaid and Waivers
    Medicaid and Government Programs
  53. Insightful Moments Video
    Medicaid and Waivers (video)
    Medicaid and Government Programs
  54. Insightful Moments - Medicaid and Government Programs - SSI and SSDI
    SSI and SSDI
    Medicaid and Government Programs
  55. New
    Insightful Moments Video
    SSI and SSDI (video)
    Medicaid and Government Programs
We are here to help

Reach Out!

To learn more about Insightful Moments please contact us at InsightfulMoments@ptcbio.com
or call 1-833-PTC-HOPE, 1-833-PTC-VIDA (Spanish).