Rett, page-7

Thanks RoyR and What a Gas. It is rare that you can get involved in an investment that has the potential to change so many lives; perhaps millions of lives if the Fragile X trial is successful and Trofinetide expands into the complete range of autism spectrum disorder. And if the trials in Traumatic Brain Injury are successful you can add millions that suffer TBI each year. Then add the devastated families of those with ASD and TBI and you are looking at an extraordinary medicine. I cannot think of another in recent history that has the potential to have such a big impact and we are part of it.


Every time I distress over bot trading that sends NEU shares down again, to put things into perspective, I reread the comments of the parents of Rett girls:

“Even though Ann was in the low dose phase we feel her behaviour changed noticeably. During the study she became very, very calm. Alertness was noticeably improved. Hand wringing and hyperventilating stopped completely. Once the test ended she reverted to former behaviour. A remarkable demonstration of the drug benefit in my mind. Imagine what full dosage for an extended period will do. I have very high hopes.”

“I was completely overcome with emotion. I couldn’t believe the words I read on my screen, “It opens not only the possibility of successful treatment of adults with Rett syndrome, but also of early interventions modifying the course of the disease.” and “exceeded the pre-specified criteria for improvement in core efficacy measures compared with placebo” It was like a dream coming true. Literally, I had dreamed of a day when there was something known to treat Rett syndrome and then on November 11th data appeared to say that might happen. I couldn’t feel my legs, I messaged with families I knew had been in the trial, with friends that had helped me make it easier for them, the world had changed and I got to be a small part of the process.


And finally, to quote Melelllan (Melinda Lancaster)

“And Neuren…. what can I say? They stand on the brink, too. Can you imagine? Being the people who save so many? I wonder if that brings the feeling of, “but we could’ve saved more” when the wheels of FDA approval seem to move so slowly. I hope not. I hope they know that those of us waiting, not just Rett parents, but Fragile X, Autism, Traumatic Brain Injury families, we’re just all so grateful for the Hope.”


Until today I was unaware of the personal and financial cost of being in the NNZ-2566 clinical trial. I found the following on a 2014 web site:

“Melinda Lancaster of Cape Cod, whose daughter Katelin, age 21, has Rett syndrome, can’t say enough good things about the way people rallied together to help Katelin get to Texas for a clinical trial which started last year. So far the trial has involved two trips, with over 40 days away from home and a cost of over $7,000 not covered by insurance. Melinda collected funds through yard sales, community organizations and online donors who read her blog. “It was a real worldwide group effort to get my child to Texas,” she says. Now there is a new organization called “Rettland” that aims to consolidate fundraising efforts for families such as Katelin’s, so that they can focus on the trial without having to worry about transportation and lodging expenses.”


For any HC posters who donated – a much overdue thank you. If additional clinical trials are needed, I think it would be a good idea for NEU investors to help those on the trial – I will be.



Mal