Dr. Emil Kakkis: Developing Novel Treatments for Rare Disorders and Connecting With Patients as Critical Partners in Research

WCG Institute Podcast

“In this episode, WCG’s President of Patient Advocacy, Steve Smith, talks with Emil Kakkis, MD, PhD, Chief Executive Officer of Ultragenyx Pharmaceuticals and President of the non-profit EveryLife Foundation for Rare Diseases organization. Known for his work in developing an enzyme replacement therapy for the rare disorder MPS I and his connection to the Ryan Foundation, Dr. Kakkis shares with us his experiences in being involved in bringing drugs to market and forming relationships with patients along the way.”

The Many Benefits of Participating in Research

Healthcare Triage Podcast

“In this episode, Aaron Carroll talks with Dr. Peter Embi about the All IN for Health program, which promotes health resources and opportunities to participate in research and clinical studies.”

Understanding Clinical Trials

Animated Pancreas Patient

“This animation explains what clinical trials are, how they are conducted, and why they are important for patients with diseases like pancreatic cancer. The animation also provides an overview of study design, eligibility criteria, informed consent, safeguards, different phases of clinical trials, and the potential benefits and potential risks of participation.”

The Clinical Trial Journey

Mayo Clinic

“Patients are waiting. That is what motivates Mayo Clinic’s researchers in their quest for better treatments and cures. Clinical trials bridge the gap between research and patient care, and help a discovery become a therapy. The clinical trial journey is how a medical intervention — such as a drug, device, or procedure — moves from an idea into patient care. From the lab to research with the help of volunteers and through review and approval for use, this animation will help you understand the biomedical research pipeline.”

What Are Clinical Trial Phases?

National Cancer Institute

“Watch to learn about the three main phases of clinical trials. Clinical trials are research studies that involve people. Understanding what they are can help you decide if a clinical trial might be an option for you or a loved one.”