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Category Archives: Orpan Drug Status
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 Ashley Winslow, clinicaltrials.gov, Gauri Srivastava, lavender, Penn Libraries, Rett syndrome clinical trials, University of Pennsylvania
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 Acadia, Dame Margaret Brimble, dendrites, Intrepid 2566, lavender and lilac, Neuren Pharmaceuticals, synaptic space, Walter Reed Army hospital
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 Anavex, Anavex 2-73, Newron, Office of Orphan Products Development, Priority Review Voucher, Rare Pediatric Disease (RPD) Designation Program, Rare Pediatric Disease Designation, sarizotan
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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 Anavex, Anavex 2-73, clinicaltrials.gov, Dr. Walter Kaufmann, FDA, homeostasis in rett syndrome, Rett syndrome clinical trials, Rett syndrome research, Rettland Foundation, Rettsyndrome.org, rettsyndrometrial.com
3 Comments
Hold the Presses-Trofinetide in the News!
The first publication of the Pediatric Trofinetide trial has hit the proverbial newsstand! Neurology.org has published “Double-blind, randomized, placebo-controlled study of troļ¬netide in pediatric Rett syndrome” ahead of print. So, it’s hotter than “hot off the press.” So, quick cut … Continue reading
Posted in Acadia, anxiety in Rett syndrome, Ataxia, Breathing issues in Rett syndrome, Communication, cure, Dr. Walter Kaufmann, hand clasping in Rett syndrome, Intrepid 2566, margaret brimble, Neuren Pharmaceuticals, Neurology.org, NNZ-2566, Orpan Drug Status, Rett Research, Rett Syndrome, Rett syndrome Awareness, rettsyndrome.org, screaming in rett syndrome, seizures in Rett syndrome, Texas Children's Hospital, Trail to a Texas Trial, Trofinetide, Trofinetide Pediatric trial, Uncategorized
Tagged * Jeffrey L. Neul, Alan K Percy, Daniel G. Glaze, George Stoms, Larry Glass, Margret Brimble, Nancy E. Jones, Neurology magazine, Neurology.org, pediatric trials, Pediatric trials results, Rett syndrome Awareness, Rett syndrome clinical trials, Rett syndrome research, Rett Syndrome Symptoms, Rett syndrome.org, Scholarly publication of pediatric trofinetide trial results, Sean Condon, Trail to a texas Trial, Trofinetide, Trofinetide Pediatric trial
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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
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 Acadia Phamaceuticals, Australia, Dr. Richard Treagus, fast track status, FDA, Fragile X, Hotcopper, Intrepid 2566, morgans financial limited, Neuren Pharmaceuticals, orphan drug designation, Pediatric voucher, Phase III trofinetide, Pitt street Research, Rett Research, Rett syndrome, Rett syndrome clinical trials, Rett syndrome.org, rettland.org, Trofinetide Pediatric trial
7 Comments
Sarizotan Trial #2 A Bit of Background
So, I was going to talk about how Sarizotan is supposed to work to help with daytime apnea this week, but I thought I’d give a bit of background on it instead. š Back in 2002-2003, Sarizotan was used in … Continue reading
Posted in anxiety in Rett syndrome, Breathing issues in Rett syndrome, Newron, Orpan Drug Status, orphan drug designation, Rett Research, Rett Syndrome, rettland.org, sarizotan, screaming in rett syndrome, Trail to a Texas Trial, Uncategorized
Tagged apnea in rett syndrome, breathing abnormalities, daytime apnea, daytime apnea in Rett syndrome, dyskenesia, EMA, FDA, Newron, orphan drug status, Parkinson's, sarizotan, Sarizotan trial
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Trofinetide #3
And on we go! So far I’ve discussed what Trofinetide is, how it came about and what it does. Great, but what are we talking for a timeline, right? Who holds the strings? Just what the heck are the steps … Continue reading
Posted in boys with Rett syndrome, breakthrough therapy, FDA, hope, margaret brimble, Neuren Pharmaceuticals, NNZ-2566, Orpan Drug Status, orphan drug designation, Rett Syndrome, rettland.org, Trail to a Texas Trial, Trofinetide, Trofinetide Pediatric trial, Trofinetide Phase III, Uncategorized
Tagged Breakthrough Therapy Designation, fast track status, FDA, Neuren Pharmaceuticals, orphan drug designation, Trail to a texas Trial, Trials, Trofinetide
20 Comments
The Next Steps Through the Desert
Wow. While time has been passing, Neuren has quietly, behind the scenes, Ā been taking step after step and gaining ground with Trofinetide. Check out their latest announcements… http://www.neurenpharma.com/IRM/ShowDownloadDoc.aspx?SiteId=386&AnnounceGuid=23a3caf0-969f-4269-824e-fe32548eefa9 http://www.neurenpharma.com/IRM/ShowDownloadDoc.aspx?SiteId=386&AnnounceGuid=89e7ec87-31e5-4aa3-b029-0b449df3189c So, to me it feels like we went from full stop … Continue reading