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 WEBEX (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 Webinars.


Most Recent Webinar – What in the World is Wrong with Me? Patients and parents of children with rare diseases learnt about one of the latest techniques for diagnosing rare diseases


Date: 30 June 2016
Time: 8 am PST / 11 am EST / 4 pm London / 5 pm Paris
See Flyer Below or Download here) –

Webinars are designed for patients with rare diseases and parents of children with rare diseases.

To Register, CLICK the LINK BELOW:


This will be 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”. Come and find out how the latest diagnostic tests can help you to reach an accurate diagnosis of your rare disease, or your child’s rare diagnosis.

We will allot a limited number of slots to Sponsor organizations. To Sponsor the Webinar, register via the above Link and indicate that you will Sponsor. Sponsors do not pay, but are asked to circulate the information about the Webinar to their patients/parents of children with rare diseases.

If you work for a Pharma organization, or a foundation, and would like to sponsor the organization financially, email LSPEID@PRDPF.ORG with SPONSOR in the subject line. Someone will contact you with more details.


Past Webinars have covered the following topics

Webinar 001 – The Seven Things You Need to Know about the Drug Development Process For Rare Diseases, Dr. Lorna Speid, PRDPF!

Webinar 002 – “Will Someone Please Tell Me What is Wrong With Me?” – Dr. Timothy McDaniel, now with TGEN

Webinar 003 – “The Rare Diseases Patient at the Center of it All” – Dr. John Whyte, FDA

Webinar 004 – “The Rare Diseases Patient at the Center of it All – Part 2”– Patient Engagement – Dr. Sangeetha Jethwa, Roche

Webinar 005 – “Natural History Studies – Making a Contribution” – Part 1 – Laying a Foundation  – Dr. Nuria Carrillo, NIH-NCATS

Webinar 006 – “Natural History Studies and Your Rare Disease Child” – Part 2 –Dr. Ann Barbier, Agios Pharmaceuticals and Professor Morton Cowan, UCSF

Webinar 007 – “What in the World is Wrong with Me?” – Amanda Balog and Stephanie DeWard from GeneDx were special guest speakers.



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. recommended.

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