Newsworthy News!

Today, like many other Rett parents, I got an e-mail from Neuren in regards to their Chairman’s Address to Stockholders. It was chock full of hopeful expectations. Of particular note:

“Finally, I would like to make some remarks in relation to the analysis of the study. A small, exploratory study such as this does not readily lend itself to conventional statistical methods more applicable to larger or pivotal trials. In recognising this fact, we chose to focus instead on quantifying a pattern of benefit across multiple outcome measures.  The data were subjected to both group-level analysis and subject-level analysis, then “stressed” by utilising a permutation test method.  This latter test is an accepted method for ascertaining the probability of observing the combined degree of clinical benefit in both the group level and the subject level analysis by chance alone, a phenomenon often referred to as a “false-positive” result. This probability was determined for our efficacy data set to be 2.3% (p=0.023).
Expert opinion on the analysis of the study and the results obtained has given us strong encouragement to explore the effect of trofinetide in a younger patient population and potentially to use a higher dose and longer duration of treatment.
We anticipate that in our upcoming meeting with the FDA we will have the opportunity to test our assumptions on study design, patient selection and dosing, as well as gain detailed input on the other remaining elements of the Rett syndrome development program.”

You can read the full report here. Clilck on the Chairman’s Address to Stockholders.

I am definitely looking forward to hearing how that meeting goes and what the next steps are. But, even more so, I am excited about a pediatric trial with a higher dose and longer duration of treatment. This is where, I believe, the greatest impact of Trofinetide can be explored. Imagine! Not trying to repair a brain in an adult, but getting to it before it needs such massive intervention! I am so hopeful about what that can mean for the younger children and their parents.

When the time comes for that trial, I hope that the families come out in droves and remember that is there to help.

Now, on to a possible new avenue for the advocacy of Rett syndrome and Trofinetide. Ann Romney and the Ann Romney Center for Neurological Diseases at Brigham and Women’s in Boston are mounting a social media campaign called 50 Million Faces- asking people (patients, family members, caregivers, friends) to share their stories about MS, ALS, Parkinson’s, Alzheimer’s and brain tumors. Well, it turns out that Trofinetide and  NNZ-2591, a close relative to Trofinetide, have the potential to be effective in treating some of these as well. (I don’t know if NNZ-2591 is a more concentrated version of Trofinetide or actually different)

From Neuren’s website:

“In large part because of the commonality of underlying pathologic processes, Neuren believes that a product which proves to be safe and effective in Rett Syndrome, Fragile X Syndrome, or TBI has good potential as a therapy in a wide range of other neurological disorders. NNZ-2566 and NNZ-2591 could be good candidates for other neurodevelopmental disorders such as Angelman Syndrome and idiopathic autism, or neurodegenerative disorders such as Parkinson’s disease and Multiple Sclerosis.”

So, on the off chance that this info has not made it’s way to the Ann Romney Center for Neurological Diseases, I’ll be writing them to let them know about it. A win for one could be a win for many-more than just 50 million, like these!-



Here’s what I wrote to Brigham and Women’s Ann Romney Center

Dear Mrs. Romney,
As a parent of a child with a neurological disorder (Rett syndrome, more prevalent than ALS) I am writing to make you aware, if you are already not so, about an experimental drug called Trofinetide and it’s close relative, NNZ-2591. These drugs, developed by Neuren Pharmaceuticals with the aid of Walter Reed Army Hospital are showing promise in the clinical and preclinical settings for treating multiple neurological disorders, including Rett syndrome, TBI, Fragile X (Clinical) and MS and Parkinson’s (Pre-clinical).

I am enclosing just two links of many that remark upon the effectiveness and encouraging results seen with these two compounds.…/Neuren-Pharmaceuticals–advance…/…/additional-neurological-condi…

There are more than 50 Million Faces affected by Neurological Diseases, Rett syndrome and Fragile X alone are more prevalent than some mentioned in this campaign. I have seen first hand (my daughter participated in the Rett syndrome clinical trial) the truly remarkable affect of Trofinetide. I believe these compounds to hold true promise in alleviating the effects of some of the most terrible neurological conditions and that your organization could, by joining the many others interested in continuing the research and development of these compounds, help change the face of the outcome of these conditions. A win for one could be a win for many.

Sincerely and respectfully,

Melinda Lancaster

This entry was posted in Ann Romney Center for Neurological Diseases, Brigham and Women's, cure, Neuren Pharmaceuticals, NNZ-2566, Rett Research, Rett Syndrome,, TBI, Trofinetide and tagged , , , , , , , , , , . Bookmark the permalink.

One Response to Newsworthy News!

  1. طفلتي راما مصابة بمرض الرت سندروم وتحتاج الي دعمكم

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