Видео ютуба по тегу Rare Disease Therapy

Increase in use of gene therapy for rare diseases

Increase in use of gene therapy for rare diseases
Focus on Rare Disease: Prime time – gene therapy for rare disorders

Focus on Rare Disease: Prime time – gene therapy for rare disorders
Gene therapy into brain helps kid with rare disease

Gene therapy into brain helps kid with rare disease
The Challenge to Develop and Commercialize Gene-Based Therapies for Rare Diseases

The Challenge to Develop and Commercialize Gene-Based Therapies for Rare Diseases
Boy with ultra-rare disease begins groundbreaking gene therapy

Boy with ultra-rare disease begins groundbreaking gene therapy
Gene Therapy: What Does It Mean for Rare Disease Communities?

Gene Therapy: What Does It Mean for Rare Disease Communities?
Gene editing saves baby with rare genetic disease

Gene editing saves baby with rare genetic disease
Rare diseases and the Bespoke Gene Therapy Consortium: Max Presa, Ph.D.

Rare diseases and the Bespoke Gene Therapy Consortium: Max Presa, Ph.D.
Understanding Biology to Investigate Potential Treatments for Rare Diseases

Understanding Biology to Investigate Potential Treatments for Rare Diseases
Challenges in Rare Disease Treatment

Challenges in Rare Disease Treatment
Promising new gene therapy treats baby with rare genetic disorder in medical first

Promising new gene therapy treats baby with rare genetic disorder in medical first
19-year-old not covered for costly treatment for rare disease

19-year-old not covered for costly treatment for rare disease
Challenges of Launching Rare Disease Therapies

Challenges of Launching Rare Disease Therapies
Pioneering Rare Disease Therapies: 2024 Rare Disease Day Webinar Highlights

Pioneering Rare Disease Therapies: 2024 Rare Disease Day Webinar Highlights
Introducing the Center for Rare Disease Therapy at UPMC Children's Hospital of Pittsburgh

Introducing the Center for Rare Disease Therapy at UPMC Children's Hospital of Pittsburgh
Rare Disease Drug Development Education Series Trailer

Rare Disease Drug Development Education Series Trailer
What Is the Future of Rare Disease Therapeutics?

What Is the Future of Rare Disease Therapeutics?
Developing customized, novel rare disease therapies: Meet Rich | The Disruptors

Developing customized, novel rare disease therapies: Meet Rich | The Disruptors
Philadelphia doctors use gene editing therapy to treat child with rare disease for the first time

Philadelphia doctors use gene editing therapy to treat child with rare disease for the first time
Jerry Vockley, MD, PhD, |Center for Rare Disease Therapy | UPMC Children's Hospital of Pittsburgh

Jerry Vockley, MD, PhD, |Center for Rare Disease Therapy | UPMC Children's Hospital of Pittsburgh