Do you have a rare disease?
Does your child have a rare disease?
PRDPF! provides invaluable information on the clinical trial and drug development process. It will be a good use of your time to take part in these Webinars. The topics that will be covered will help you to better understand the drug development process, and engage with it.
The sessions will be hosted via GoToMeeting (similar to SKYPE) and will allow patients to interact with Dr. Speid and guest speakers, as well as each other in a non intimidating way. You will be able to sit at your computer/laptop, use a tablet or even your phone, to connect to these informational Webinars.
Webinars are designed for patients with rare diseases and parents of children with rare diseases.
Webinar 7 was a very helpful and insightful look at the techniques used to diagnose causes of rare diseases. You’ve heard the phrase -“It’s all in the genes”. Patients and parents found out how the latest diagnostic tests can help them to obtain an accurate diagnosis of their rare disease, or obtain a diagnosis for their child.
Past Webinars have covered the following topics
Resources for Patients and Parents
Clinical Trials: What Patients and Healthy Volunteers Need to Know
The Book Clinical Trials: What Patients and Healthy Volunteers Need to Know, published by Oxford University Press, is the source text for the information that is covered in the Webinars. For a deeper understanding of the clinical trial and drug development process, this book is recommended. You can purchase the book from the publisher by following the link below.
https://global.oup.com/academic/product/clinical-trials-9780199734160?cc=us&lang=en&
Diagnosis: Rare Disease
“Rare Disease” the diagnosis that no parent ever wants to hear. “This book gives a heartbreaking exposé of what life is like for families that have a member with a rare disease,” explains Carolyn Paisley-Dew, ISMRD Board Member.
Publication By Dr. Lorna Speid
Dr. Lorna Speid, President of PRDPF! spoke at the American ASCPT Meeting in 2016. Her presentation was published in the ASCPT’s peer reviewed journal. You can find the reference below. In the publication, Dr. Speid provides a summary of the challenges faced by patients with rare diseases.
Speid, L. (2016), Don’t Do Different Things – Do Things Differently! Drug Development in Rare Diseases: The Patient’s Perspective. Clin. Pharmacol. Ther., 100: 336–338. doi:10.1002/cpt.403.
Rare Like Us: From Losing My Dad to Finding Myself in a Family Plagued By Genetic Disease
Taylor Kane was a daddy’s girl from the moment she was born, smiling and cooing whenever her father was around and refusing to sleep until he held her in his arms. But shortly after she turned three years old, the unthinkable happened. Her father was diagnosed with a rare, genetic disease for which there was no cure. It wasn’t long before he began to experience a number of bizarre and frightening symptoms, and young Taylor watched helplessly as the disease ravaged his body and mind, transforming him into a shell of the father she once knew