Crush Rett Syndrome

The 2nd adult patient dosed in Taysha's gene therapy trial is reporting positive results!

“The two adult patients dosed with TSHA-102 have different mutations in their MECP2 gene that manifest in different phenotypes and clinical severity. Following treatment, both patients experienced improvement in key clinical domains impacting activities of daily living, including breathing dysrhythmia, autonomic function, socialization, and gross and fine motor skills. Both patients display significantly reduced breathing dysrhythmia, with less breath holding spells and infrequent hyperventilation, improved limb perfusion and vastly improved interest in social communication and activities. In addition, the first patient experienced sustained and new improvements, with restored movement in her legs and the gained ability to sit unassisted for up to 15 minutes for the first time in over a decade. Further, her hand function improved with the gained ability to grasp objects with her non-dominant hand and transfer them to her dominant hand for the first time since infancy. Following treatment, the second patient’s posture, gait and stability improved, resulting in straighter posture and smoother movements when walking. Her hand stereotypies also improved for the first time since regression at age three: she now displays less forceful hand wringing and her hands are often open and relaxed, providing new opportunities for fine motor skill learning. In addition, her seizures are much less frequent. I’m encouraged by the early positive signals and consistent improvement seen in both patients following treatment.”

Taysha Gene Therapies Reports Third Quarter 2023 Financial Results and Provides Corporate and Clinical Updates | Taysha Gene Therapies Data from first adult patient in REVEAL Phase 1/2 trial showed TSHA-102 was well-tolerated with no treatment-emergent SAEs as of 20-week assessment with sustained improvement across key efficacy measures and new improvement in R-MBA, PGI-I and hand function, a hallmark characteristic of Rett

Thank you for following along this month! We will continue to post updates about the progress of Rett syndrome research the human clinical trials. Stay strong and continue the fight to crush Rett syndrome!

Did you know that there are currently two different approaches being studied for a cure? Neurogene is also recruiting for clinical trials to test out their version of a cure. So many exciting possibilities in the very near future.

2023 saw the first human clinical trials begin for Taysha’s gene therapy and the first two patients dosed. Initial reports are extremely promising of a reversal of symptoms. Our cure is within reach for the first time ever!

People born with Rett Syndrome and their families were taught how to manage symptoms and prevent complications, but now there is hope for a CURE!

Human trials started THIS YEAR. For the first time, a REVERSAL of symptoms is on the horizon

Rett Syndrome causes more physical disability than intellectual, so presume competence. Talk to them even if you don't see an expected response. Anyone with a physical disability should be presumed to be intellectually competent.

Aging is difficult for everyone. Many parents are fortunate to have children that can help their parents as they become unable to care for themselves. Parents of children with Rett Syndrome have to take the time to care for themselves so that they can care for the child for as long as possible.

When many parents start to see their children leave home for jobs or college, parents of Rett Syndrome children have to figure out what the future holds for themselves and their child. Loving, safe, and consistent care can be hard to find and financially draining.

While their friends feel like they are constantly driving their teenagers to various activities, parents of Rett Syndrome children find themselves more and more unable to participate in social events. Seizures, wheelchair accessibility, anxiety, and simply exhaustion can cause this. But also, friends and family may stop inviting them to their gatherings because they aren't able to or prepared to make the accommodations needed.

Many children develop scoliosis during their growth spurt years. Rett Syndrome doesn't shield a child from other disorders or diseases. But, many children with Rett Syndrome develop very aggressive scoliosis advancing from just a few degrees of curve to as much as 90 degrees or more in a short period of time. Without correction, this can be fatal.

After years of being your child's sole caregiver, it can be exciting and scary to send your child to school that first day. But when your child can't speak, can't defend themselves, may not be able to walk, and likely is in a diaper, it is a cause for great anxiety. How will a stranger know what your child needs? And when they get home, how will you know if they were treated well and their needs were met?