Category Archives: Anavex

IRSF Scientific Meeting, Daybue and More

Posted on June 19, 2023 by melelllan

The International Rett Syndrome Foundation (IRSF) held its Scientific Meeting on June 5-7, featuring presentations from Acadia, Taysha, Neurogene, and NeuroTec. Anavex did not present as they had no new information to share. Acadia presented LILAC data and introduced a new Real World Efficacy study called LOTUS. Unfortunately, any data presented at the meeting is not publicly available, but a summary will be provided by IRSF at a later date. Dr. Jim Youakim gave a historical presentation about Trofinetide’s development, with an emphasis on the LAVENDER study and challenges Acadia faced during the pandemic. Continue reading →

Posted in Acadia, Anavex, Daybue, IRSF, LAVENDER TRIAL, Neuren Pharmaceuticals, Rett Syndrome, Trail to a Texas Trial, Uncategorized | Tagged , , , , , , | Leave a comment

ASCEND 2022!

Posted on May 11, 2022 by melelllan

After a VERY long hiatus, the annual/biannual Rett syndrome Family Conference came back with a BANG! The backdrop being the incredible, but entirely too large, Gaylord Resort, Nashville. Thank you to IRSF for putting together such a massive and comprehensive … Continue reading →

Posted in Acadia, Anavex, Ascend Rett syndrome conference 2022, boys with Rett syndrome, clinicaltrials.gov, Communication, David Clements, Dr. Helen Leonard, Dr. Walter Kaufmann, FDA, fund-raising for rett syndrome, hope, Huda Zoghbi, Kevin Black, LAVENDER TRIAL, Raising a Hand, Rett Research, rettsyndrome.org, Telethon for Kids, Trail to a Texas Trial, Trofinetide, Trofinetide Phase III, Uncategorized | Tagged , , , , , , , , , , , , , , , , , | Leave a comment

Rett syndrome and Pandemic Don’t Mix

Posted on February 6, 2022 by melelllan

I was going to write about the success of the Anavex trial, but it will have to wait for another day; it is simply impossible to try and explain it when Rett syndrome is screaming at me- I’m NOT going … Continue reading →

Posted in Anavex, Anavex 2-73, Uncategorized | Leave a comment

A Challenge Accepted! ;)

Posted on November 23, 2019 by melelllan

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

A Day with Acadia- Part 1: Awareness

Posted on October 30, 2019 by melelllan

This is my last post of Rett syndrome Awareness month. I had hoped to do more, but Rett syndrome made sure a good amount of awareness was happening right at home, but this was going to be the last either … Continue reading →

Posted in Acadia, Anavex, Anavex 2-73, Communication, Dame Margaret Brimble, fund-raising for rett syndrome, gp2c, hope, ketamine, kyphosis, Neuren, Neuren Pharmaceuticals, Newron, NNZ-2566, NNZ-2566 trial, Remarkable Rett-a podcast series, Rett Research, Rett Syndrome, Rett syndrome Awareness, rettsyndrome.org, RSRT, sarizotan, service dog, Talking, Trail to a Texas Trial, Trofinetide, Trofinetide Phase III | Leave a comment

Close is Too Late for Too Many

Posted on August 24, 2019 by melelllan

As we approach the start of the Trofinetide trial, I know that it is going to go by in a blink.  And, there are other trials in different stages of development, each holding some parent’s hope for their child- improvement … Continue reading →

Posted in Acadia, Anavex, Avexis, boys with Rett syndrome, cure, Death in Rett syndrome, grief, Neuren Pharmaceuticals, Rett Research, Rett Syndrome, Rett syndrome Awareness, tracy chapman, Trail to a Texas Trial, Trofinetide Phase III | Leave a comment

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

Posted on April 12, 2019 by melelllan

…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

Posted on February 28, 2019 by melelllan

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

Right to Try-Walking the Tightrope

Posted on June 2, 2018 by melelllan

Yesterday, the president, signed the Right to Try Bill into law. But, what does that really mean for the majority of patients suffering from TERMINAL illness? I’ll try to break it down as best as possible from what I know … Continue reading →

Posted in Anavex, cure, FDA, fund-raising for rett syndrome, hope, Rett Research, Right to Try Law, Trofinetide, Trofinetide Phase III, white house | Tagged , , , , | 3 Comments

What do Halloween and Rett syndrome have in Common? Superheroes.

Posted on October 31, 2017 by melelllan

Today is the last day of October. Another year gone by from one Awareness Month to the next and still no treatment. But this is not a humdrum post BECAUSE treatment is now within sight. Each year in October superhero … Continue reading →

Posted in Anavex, Anavex 2-73, boys with Rett syndrome, cure, fund-raising for rett syndrome, Neuren Pharmaceuticals, Newron, Rett Research, Rett Syndrome, Rett syndrome Awareness, sarizotan, Trofinetide, Trofinetide Phase III, Uncategorized | Tagged , , , , , , , , , , , , | Leave a comment