Local Company Develops Therapies For Rare Diseases

Local company develops therapies for rare diseases

Chronicle 5 WCVB

The Challenge to Develop and Commercialize Gene-Based Therapies for Rare Diseases

RDCA-DAP: Shortening the Timeline for Developing New Treatments for Rare Diseases

National Organization for Rare Disorders (NORD)

The challenges of developing treatments for rare diseases

BioIndustry Association - BIA

AGTC - Genetic Therapy For Rare Diseases

HPR

Medical Pioneer: First Patient Receives Unique Rare Disease Therapy Developed at UC San Diego

UC San Diego Health

How Rare Disease Treatments Get Approved

Grand Rounds: Developing Gene Therapies for Ultra-rare Diseases in the Academic Setting

MassGeneralHospital

How To Crowdfund For Rare Diseases With Milaap?

Challenges in developing gene therapies for rare diseases & CNS disorders

Specialist healthcare communications: the challenges for cell and gene therapies and rare diseases

Emil Kakkis, M.D., Ph.D. - Developing Treatments for Rare Diseases

Sanford Burnham Prebys

Natural History Studies for Rare Diseases

U.S. Food and Drug Administration

300 million dollars in federal funding to develop treatments for rare diseases

Rare Disease Drugs and Market Implications

RTI Health Solutions

UT Southwestern creates customized treatments for rare diseases

UT Southwestern Medical Center

Webinar Teaser | Gene Therapies for Rare Diseases: Advancing Research with Humanized Models and AI

Challenges in Rare Disease Treatment

Alliance for Patient Access

Gene Therapy for Rare Disorders | UPMC Children's

childrenshospgh

Overcoming manufacturing issues in gene therapies for rare diseases