Published Dec 10, 2024

Rare Disease, Big Impact with Matt Wilsey

Matt Wilsey narrates his inspiring journey from confronting his daughter's rare genetic disorder, NGLY1 deficiency, to founding Grace Science, a biotech company aiming for a cure. The episode explores the challenges and triumphs of navigating FDA regulations, strategic biotech partnerships, and the cutting-edge potential of gene therapy in combating rare diseases.

Episode Highlights

Topics covered

Popular Clips

Questions from this episode

- How does gene testing work?
  Asked by 6 people
- What is gene therapy?
  Asked by 5 people
- What is the history of genetics in relation to the episode George Church, PhD: Rewriting Genomes to Eradicate Disease and Aging and the clip Human Genome Project?
  Asked by 3 people
- What is gene therapy in the context of the episode The Power of a Platform Company with Josh Mandel-Brehm and the clip Gene Therapy Insights?
  Asked by 2 people
- How are rare diseases being treated as discussed in the episode John Crowley Interview | The Tim Ferriss Show (Podcast) and the clip Impact Through Altruism?
  Asked by 1 person
- How is gene testing conducted?
  Asked by 1 person
- How is gene therapy delivered?
  Asked by 1 person

Episode Highlights

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Dexa/Raising Health

Rare Disease, Big Impact with Matt Wilsey

Topics covered

Popular Clips

Genome Sequencing Evolution

Gene Therapy Advances

Navigating FDA Challenges

Diagnostic Odyssey

Gene Discovery Journey

Unlocking NGLY1 Potential

Navigating FDA Relationships

Business for Change

Questions from this episode

How does gene testing work?

What is gene therapy?

What is the history of genetics in relation to the episode George Church, PhD: Rewriting Genomes to Eradicate Disease and Aging and the clip Human Genome Project?

What is gene therapy in the context of the episode The Power of a Platform Company with Josh Mandel-Brehm and the clip Gene Therapy Insights?

How are rare diseases being treated as discussed in the episode John Crowley Interview | The Tim Ferriss Show (Podcast) and the clip Impact Through Altruism?

How is gene testing conducted?

How is gene therapy delivered?

Episode Highlights

Diagnostic Odyssey

Biotech Venture

Regulatory Navigation

Related Episodes

Solving Medical Mysteries in the World of Rare Disease

The Power of a Platform Company with Josh Mandel-Brehm

Drag & Drop Genome Editing

A Fatal Inheritance with Lawrence Ingrassia

CRISPR by Design with Benjamin Oakes

Adapting Biopharma to AI with Greg Meyers

Drugging the Undruggable with Greg Verdine

The Science and Supply of GLP-1s with Brooke Boyarsky Pratt

Fireside Chat with Sean Duffy

Protecting the Golden Age of Medicine with John Crowley

Network Medicine with Jonathan Swerdlin and Mark Hyman

The Story of Schizophrenia

The Power of Drug Discovery with Philip Tagari

Adapting Biopharma to AI: A 2024 Update with Greg Meyers

Journal Club: Why do only some people get severe COVID-19?

Rare Disease, Big Impact with Matt Wilsey

Topics covered

Popular Clips

Questions from this episode

Episode Highlights

Diagnostic OdysseyMatt Wilsey shares the harrowing journey of diagnosing his daughter Grace with NGLY1 deficiency, a rare genetic disorder. His determination led to the founding of Grace Science, a biotech startup focused on finding a cure.

Diagnostic Odyssey

Biotech Venture

Regulatory NavigationMatt Wilsey shares insights into navigating FDA interactions and developing approval strategies for gene therapies. His work focuses on advancing treatments for rare diseases, emphasizing collaboration and innovation.

Regulatory Navigation

Related Episodes