Challenge Accepted!

Leap of Faith

From: Trail to a Texas Trial It hasn’t happened before. I was given a MISSION, if I chose to accept it, to explain some new developments in regards to Sarizotan (Newron) and Anavex 2-73 (Anavex) receiving FDA Rare Pediatric Disease Designation: “Mel Lancaster have you posted about these announcements or am I just behind on […]

A Day with Acadia- Part 2: Assurance

One thing Bill Keller said that really resonated with me because of the sincerity and sense of duty that he said it with- (paraphrasing here) “I met Steve Kaminsky and he told me that since this drug (Trofinetide) existed it was our responsibility to get it to families.”

Hot off the Presses- The Horses are Outta the Gate!

Well yesterday’s post WAS going to be the last one this month but then I got an email from Acadia- “Hot off the Presses” it said and then this announcement- ACADIA Pharmaceuticals Initiates Phase 3 Pivotal Study of Trofinetide in Rett Syndrome, a Rare Neurodevelopmental Congenital CNS Disorder  The tears started around the word “initiates” […]

A Day with Acadia- Part 1: Awareness

This Blog is hosted on behalf of Mel Lancaster, whose original blog was silenced. I like to end this month on an up note and that presented itself when I was invited to speak at Acadia in San Diego. Bill Keller, Executive Director; Patient Advocacy and Industry Relations, of Acadia wanted to do a Rett syndrome Awareness […]

Rett syndrome Awareness Month 

Hi Im Carys

Why it’s important. The only way we will truly disrupt socially, is when we all share the exact same thing. This is a hefty read, here’s a summary. I will talk about : Inclusion, it’s more than just people living with Rett How going viral made a charity $50 Million New Diagnoses Bad information Good […]

My Rett journey

Don;t be their voice, amplify it

I wish that I knew what I know now. When I was younger. Rett syndrome is a better diagnosis now than it was a decade ago. I’ve been on this Rett trip for a while now, and my outlook has changed considerably throughout this journey. Volumes of studies, research and hard work from a fantastic […]

Revolution

This awareness month, Army of Us has been all about Revolution. We’ve shared awareness images with our take on famous propaganda posters, created profile flags and made timeline posters. But what do we mean when we talk about revolution in the context of all of us living with Rett syndrome? A revolution often happens when […]

Still water

 Your child is not “retarded”. This is very close to my heart, every day I read comments from families about the intelligence (or lack thereof) of people living with Rett syndrome. I feel I need to rectify what is being said. I know it’s not their fault, families (and friends) get their information from people […]

The Trofinetide trial is important to Rett syndrome.

We now know what Rett is, what causes it, where it’s located. We have proven it’s reversible and for the very first time, we potentially have a treatment that could profoundly impact the disorder. Crucially, improvements were shown after less than 2 months. Because of the way Trofinitide works, we could expect to see continued […]

Boys with Rett syndrome

Boys with Rett syndrome have 1 of 2 other disorders which allow for a Rett syndrome diagnosis Klinefelter syndrome (Also known as 47 or XXY) Somatic mosaicism This is because Rett syndrome is a clinical diagnosis and a clinical diagnosis for boys can only be given if 1 of these conditions is met. A clinical […]