Category Archives: FDA

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

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

Trofinetide Series-Part 2: LAVENDER-Study Description and Study Design

Alrighty then! Time to start breaking down the information on ClinicalTrial.gov. It’s going to take a while to get through all the info, however I will try to keep in concise and understandable. The key, here, is that the more … Continue reading

Posted in Acadia, clinicaltrials.gov, FDA, Neuren Pharmaceuticals, Orpan Drug Status, Rett Syndrome, Trail to a Texas Trial, Trofinetide, Trofinetide Phase III | Tagged , , , , , , | 2 Comments

Trofinetide Series-Part I: Words you Need to Know

So, over the course of the next few weeks, I’m going to be doing a series of posts to catch you all up to speed on the Trofinetide trial, which for many of you seems to be going at a … Continue reading

Posted in Acadia, boys with Rett syndrome, Dame Margaret Brimble, FDA, Intrepid 2566, Neuren Pharmaceuticals, NNZ-2566, NNZ-2566 trial, Orpan Drug Status, Rett Research, Rett Syndrome, TBI, Trail to a Texas Trial, Trofinetide, Trofinetide Pediatric trial, Trofinetide Phase III | Tagged , , , , , , , | 1 Comment

A Challenge Accepted! ;)

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 … Continue reading

Posted in Acadia, Anavex, Anavex 2-73, FDA, Neuren, Neuren Pharmaceuticals, Newron, Orpan Drug Status, sarizotan, Trail to a Texas Trial, Trofinetide | Tagged , , , , , , , | Leave a comment

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

I Love Someone Rare

Today is Rare Disease Day 2019 From the NIH website: What is a rare disease? In the United States, a rare disease is defined as a condition that affects fewer than 200,000 people. This definition was created by Congress in … Continue reading

Posted in Acadia, Anavex, Anavex 2-73, Avexis, FDA, Neuren Pharmaceuticals, Orpan Drug Status, orphan drug designation, Rett Research, Rett Syndrome, rettsyndrome.org, RSRT, Trail to a Texas Trial, Trofinetide, Uncategorized | Leave a comment

The Next Steps Up the Mountain

Neuren Pharmaceuticals and Acadia have announced more details about the upcoming Phase III trial. Read about them HERE. Two very important blurbs stick out: “The Phase 3 study will be followed by an open label extension study in which all … Continue reading

Posted in Acadia, FDA, Neuren Pharmaceuticals, Rett Research, Rett Syndrome, Rett syndrome Awareness, rettland.org, rettsyndrome.org, Trail to a Texas Trial, Trofinetide, Trofinetide Phase III | Leave a comment

All is Not Quiet on the Trofinetide Front #2; A Balancing Act

* Warning* Looooong post! If you aren’t interested in financial aspects and just want an update on logistics that have affected the timeline, skip to the second half- there’s also a link to a new update from Acadia, the pharmaceutical … Continue reading

Posted in Acadia, cure, FDA, Neuren Pharmaceuticals, Orpan Drug Status, orphan drug designation, Rett Research, Rett Syndrome, rettland.org, rettsyndrome.org, TBI, Trail to a Texas Trial, Trofinetide, Trofinetide Pediatric trial, Trofinetide Phase III | Tagged , , , , , , , , , , , , , , , , , , , | 7 Comments