T-20 Musing: Parents’ Helping Hands Posted on February 22, 2023...

T-20 Musing: Parents’ Helping Hands
Posted on February 22, 2023 by melelllan

I often see parents who comment on social media that they feel disappointed, or defeated, etc. because they were unable to participate in a clinical trial. But, it takes all types of participation for a trial and ultimate approval (hopefully!!) of a drug, say…such as Trofinetide.

Waaay back in 2022, almost a year to the day that the FDA will announce whether Trofinetide will be approved, IRSF and RSRT, along with multiple co-hosts and moderators, held and Externally-led Patient Focus Drug Development meeting.

This meeting was a day-long, two-part, intensive question-and-answer session with one focus- educating the FDA on Rett syndrome. Parents/caregivers were given the opportunity in advance to ask questions and were able to call in during the program during specific times/topics. 755 people participated in this webinar!

Several parents provided recorded statements about specific aspects of life with Rett syndrome, for which I admire them a great deal. One such person being Jack Kuwik, a dear Facebook friend, who has since passed away and will not be here to see the day that is coming. This grieves me a great deal, but I hope that wherever his soul may be that he will feel the communal joy and relief that his contributions over the years have helped bring to fruition.

The Voice of the Patient Report was prepared and submitted on behalf of the Rett Syndrome
Research Trust (RSRT) and the International Rett Syndrome Foundation (IRSF) by: Monica Coenraads,
CEO, RSRT; Jana Von Hehn, PhD, Chief Scientific Officer, RSRT; Tim Frank, Chief Marketing and
Development Officer, IRSF; Melissa Kennedy, CEO, IRSF; Dominique C. Pichard, MD, Chief Science
Officer, IRSF; Chrystal Palaty, PhD, medical writer.


Consulting Partners include James Valentine, Esq. and Larry Bauer, RN, MA, from Hyman, Phelps &
McNamara, P.C.


RSRT and IRSF contracted with Chrystal Palaty, PhD., from Metaphase Health Research Consulting Inc.for assistance in writing this report.



*(taken directly from page 3 of this report)

I recommend anyone interested in Rett syndrome read this report. For new parents and their providers, it would be an invaluable resource. For the FDA it becomes THE go to resource about parental/caregiver concerns when considering new drugs, therapies, equipment, etc. for Rett syndrome.

Why do I bring it up? Because of YOU- the parents, caregivers, relatives, and friends who participated in this nearly unprecedented meeting- one of the steps often needed when considering drug approval was not necessary, no doubt speeding up the process, even if by just a bit. Every bit counts! So, pat yourselves on the back and thank each other for this helping hand.

T-20!