Category Archives: Uncategorized

Rett syndrome Independence Day Gets (hopefully) a Little Closer! Trofinetide Reopens- An Interview with Dr. Jim Youakim

On Saturday June 27, I had the pleasure of speaking with Dr. Jim Youakim, Vice President of Research and Development, to get an update on the trofinetide trial and it’s recent reopen. Bill Keller, Executive Director of Patient Advocacy, joined … Continue reading

Posted in Acadia, clinicaltrials.gov, FDA, Neuren Pharmaceuticals, Rett Research, Rett Syndrome, Trail to a Texas Trial, Trofinetide, Trofinetide Phase III, Uncategorized | Tagged , , , , , , , | Leave a comment

Even Still, We Must Reach for the STARS

Hyperventilation, daytime apnea, breath-holding– whatever name you call it by, it’s a horrific breathing pattern that severely impacts the quality of life of many of our children and their families. In 2016, Newron Pharmaceuticals undertook the STARS trial, an international … Continue reading

Posted in clinicaltrials.gov, Newron, orphan drug designation, rettsyndrome.org, sarizotan, Trail to a Texas Trial, Uncategorized | Tagged , , , , , , | Leave a comment

Covid-19 and Rett Syndrome

This is a scary time for everyone on the planet. Because I was asked by someone in a country far away, I am posting links for those who may or may not have access to all the resources us in … Continue reading

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Tips for Topsy Turvey Times- App review #1

Ok, we’re all in a boat. There’s a storm outside and we all need to bail! So let’s bail together!! We can all do our own little part to help everyone get through this! PLEASE EMAIL ME m3ml@hotmail.com or through … Continue reading

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Trofinetide Series Part 3-LAVENDER- Arms and Interventions; Outcome Measures-Primary AKA a Leap Toward Proving Efficacy

Deep breath everybody. This is starting to get into the nitty gritty of what the hopes are for Trofinetide and how success will be measured. This can get complicated and I will try my very best to make it easy … Continue reading

Posted in Acadia, anxiety in Rett syndrome, boys with Rett syndrome, clinicaltrials.gov, Dame Margaret Brimble, FDA, Neuren Pharmaceuticals, NNZ-2566, NNZ-2566 trial, Rett Research, Rett Syndrome, screaming in rett syndrome, Trofinetide, Trofinetide Pediatric trial, Trofinetide Phase III, Uncategorized | Tagged , , , , , , , | Leave a comment

A Crystal of Happiness and Hope

There is a poem I love. It serves to remind me that we are all entitled to the “Pursuit of Happiness” but also that you must learn to be content with any pieces you find because, in truth, no one … Continue reading

Posted in hope, Poetry, Rett Syndrome, Rett syndrome Awareness, Trail to a Texas Trial, Trofinetide, Trofinetide Phase III, Uncategorized | Tagged , | 2 Comments

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 … Continue reading

Posted in Acadia, Dame Margaret Brimble, hand clasping in Rett syndrome, margaret brimble, Neuren, Neuren Pharmaceuticals, Rett Research, Rett Syndrome, Rett syndrome Awareness, rettsyndrome.org, Trail to a Texas Trial, Trofinetide Phase III, Uncategorized | Leave a comment

20 Years in Oz

Originally posted on Trail To A Texas Trial:
I saw a writing contest advertised on Facebook, the very next ad under it was something to do with the Wizard of Oz…and so I was inspired. I had hoped to get…

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Rett Syndrome is Such a Trial… #2 Ketamine

Many parents can attest to the fact that after a procedure requiring anesthesia our children have a decrease in Rett related symptoms. For Katelin, this was a drastic decrease in behavioral issues. My other children would often say… Can we … Continue reading

Posted in Breathing issues in Rett syndrome, FDA, ketamine, Rett Research, Rett Syndrome, Rettland Foundation, rettland.org, RSRT, Trail to a Texas Trial, Uncategorized | Tagged , , , , , , , , , | 2 Comments

Rett Syndrome is Such a Trial… #1 Anavex 2-73

…and 2019 looks to change that up a bit- Rett Syndrome, so many trials! According to the most recent GP2C newsletter (GirlPower2Cure)there will be at least 7 trials this year, currently in various stages. This is SO exciting! What does … Continue reading

Posted in Anavex, Anavex 2-73, cure, Dr. Walter Kaufmann, FDA, gp2c, hope, Orpan Drug Status, Rett Research, Rett Syndrome, Rett syndrome conference 2016, Rettland Foundation, rettland.org, rettsyndrome.org, Trail to a Texas Trial, Uncategorized | Tagged , , , , , , , , , , | 3 Comments