Gene Therapy: What Does It Mean for Rare Disease Communities?

(28:8)

What you need to know about the Ultragenyx gene therapy trial for Wilson disease

(14:56)

Ultragenyx is building a gene therapy plant

(2:12)

Ultragenyx Ventures into Gene Therapy for Wilson Disease

(6:32)

Ultragenyx CEO talks the company's path to profitability in developing drugs for rare diseases

(3:38)

Gene Therapy: A Conversation With Dr Benke \u0026 Ultragenyx

(1:27:1econd)

Ultragenyx: Built to be Rare

(2:48)

Ultragenyx Pharmaceutical - Dr Zoe Farrington - Gene Therapy

(16:44)

Ultragenyx

(15:45)

The Gene That Makes the Ketogenic Diet Dangerous

(10:15)

Life with GSDIa: Jonah’s Story

(4:1econd)

Cramer: This biotech stock could be worth over $100 billion on an FDA approval

(5:19)

Developing Treatments at Ultragenyx

(28:3)

Gene Therapy 101 with Dr. Fred Askari

(10:36)

2024 ASF Family Conference - Pharma and Biotech Industry Update

(48:5)

Current Policy Challenges Impacting Biotechnology Innovation

(48:36)

GenScript's Solution for Advancing the Development of CRISPR Gene Therapy with Live Q\u0026A

(40:39)

Life with LC-FAOD: Tasia’s Story

(3:48)

Gene therapy

(1:7:20)

Ultragenyx Pharmaceutical

(14:54)

Ultragenyx Gene Therapy Facility Milestone Celebration

(2:12)

Update on Ultragenyx's Sanfilippo syndrome Type A Gene Therapy program (UX111) - March 26, 2024

(1:19:38)

Ultragenyx Pharmaceutical Inc.: Overcoming Challenges

(23:47)

Rare Bootcamp: Empowering Rare Disease Warriors for the Battle

(1:55)

What You Need To Know About The Ultragenyx Gene Therapy Trial For Wilson Disease

(14:56)

Ultragenyx - Preclinical Evaluation of AAV9-CDKL5 Gene Therapy in Models of CDKL5 Deficiency Disorde

(28:5)

Ultragenyx Pharmaceutical Updates: Understanding Accelerated Approval and Path to Access

(22:44)

Ultragenyx DTX401 Update

(54:4)

2023 Ultragenyx Update

(10:59)

Ultragenyx - Kakkis

(25:4)