Category Archives: rettsyndrome.org

Where do we go From Here? Part I

Posted on August 10, 2022 by melelllan

Last week, the International Rett syndrome Foundation (IRSF) held a webinar with Dr. Dominique Pichard, CSO, IRSF and Mr. James E. Valentine, Esq., HPM aptly named, “What Comes Next? The FDA Regulatory Process from NDA to FDA decision and beyond.” … Continue reading →

Posted in Acadia, clinicaltrials.gov, Dame Margaret Brimble, FDA, International Rett syndrome Foundation, Intrepid 2566, LAVENDER TRIAL, Neuren Pharmaceuticals, NNZ-2566, NNZ-2566 trial, orphan drug designation, Rett Research, Rett Syndrome, rettsyndrome.org, TBI, Trail to a Texas Trial, Trofinetide Pediatric trial, Trofinetide Phase III | 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

A New Acronym for GLOBAL Participation!!

Posted on January 29, 2022 by melelllan

This week, I and 116 other participants engaged in a virtual presentation of a pre-FDA EL-PFDD. What a mouthful! So, what the heck does THAT all mean. Well, it means that on March 11, 10-3pm Eastern time I and ANYONE … Continue reading →

Posted in FDA, Rett Syndrome, rettsyndrome.org, RSRT | Leave a comment

A Chat with James Shaw of Neuren

Posted on January 6, 2022 by melelllan

Talking with James Shaw, Vice President, Clinical & Regulatory Operations, is like talking with an old friend. He has been there every step of the way through Katelin’s journey with Trofinetide and his dear heart is so invested in our children. The few times … Continue reading →

Posted in Acadia, Dame Margaret Brimble, Intrepid 2566, LAVENDER TRIAL, Neuren Pharmaceuticals, Rett Syndrome, rettsyndrome.org, TBI, Trail to a Texas Trial, Trofinetide, Trofinetide Phase III | Tagged , , , , , | 4 Comments

A Chat with Dr. Pichard of IRSF

Posted on December 20, 2021 by melelllan

Disclaimer: Unless specifically stated, the possible benefits down the line are my opinion alone. As one might imagine, I and other parents have a ton of questions since the results of the Trofinetide Phase III came out. One of the … Continue reading →

Posted in Acadia, anxiety in Rett syndrome, Breathing issues in Rett syndrome, clinicaltrials.gov, Communication, Daffodil trial, Dame Margaret Brimble, Dr. Helen Leonard, LAVENDER TRIAL, Neuren Pharmaceuticals, Rett Research, Rett Syndrome, rettsyndrome.org, screaming in rett syndrome, Talking, Trofinetide Phase III | Tagged , | Leave a comment

Rett syndrome Awareness Month #4 Helping Hands/Fundraisers Galore

Posted on October 4, 2021 by melelllan

One of the mainstays of this month is fundraising! From small pumpkin painting ones and the ever popular purple fingers and toes nail polish ones, to jewelry and other small businesses holding parties with a portion going to a charity … Continue reading →

Posted in David Clements, fund-raising for rett syndrome, gp2c, Kevin Black, Raising a Hand, Rett Research, Rett Syndrome, Rett syndrome Awareness, rettsyndrome.org, Trail to a Texas Trial | Tagged , , , , , , | Leave a comment

October Pandemic

Posted on October 1, 2020 by melelllan

It’s been nearly three months since I’ve put my pen to paper here, on this blog I love. The world is being ravaged by a pandemic and every day is a struggle to get by, to find some normalcy, some … Continue reading →

Posted in anxiety in Rett syndrome, Ataxia, boys with Rett syndrome, Breathing issues in Rett syndrome, Communication, Death in Rett syndrome, Education, grief, hand clasping in Rett syndrome, Pain in Rett syndrome, Rett Syndrome, Rett syndrome Awareness, rettsyndrome.org, screaming in rett syndrome, Trail to a Texas Trial | Tagged , | 1 Comment

ACADIA Talks Trofinetide and Covid-19: An Interview with Dr. Jim Youakim

Posted on May 7, 2020 by melelllan

Recently, I was given the incredible opportunity to interview Dr. Jim Youakim, Vice President of Research and Development. This was a bit out of my wheelhouse, but he and Bill Keller, Executive Director of Patient Advocacy, were very gracious in … Continue reading →

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

Even Still, We Must Reach for the STARS

Posted on May 4, 2020 by melelllan

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

An Update on Trofinetide Coming Soon to a Blog Near You!

Posted on April 13, 2020 by melelllan

This past weekend, I had the great honor of being chosen to interview  Dr. Jim Youakim. Dr. Youakim heads up the Trofinetide trial from Princeton. I thought I’d give you a preview of his qualifications and some of his thoughts … Continue reading →

Posted in Acadia, Neuren Pharmaceuticals, Rett Research, Rett Syndrome, rettsyndrome.org, Trail to a Texas Trial | Tagged , , , , , | 1 Comment