Rett In The News

I’m proud of the work we have done at my production company, Peak Curiosity, and with the charitable advocacy work we do at Magnolia’s Hope on behalf of my daughter who suffers from Rett Syndrome.

Taysha Gene Therapies announces the publication of new preclinical data for their Rett Syndrome program, TSHA-102.

Farmingdale pharmaceutical firm DepYmed is no stranger to capital investments – but a new infusion might be enough to push the rare-disease innovator over the commercialization line.

In mouse models of Rett Syndrome intensive training in young mice staved off symptoms of the disorder suggesting that perhaps the same might be true in people.

UConn professor of physiology and neurobiology Daniel Mulkey has received a $2.1 million grant from the NIH to investigate the cellular and molecular basis for disordered breathing in Rett syndrome. This project is part of a collaboration with Michelle Olsen, associate professor of neurobiology ...

UVA’s Sanchita Bhatnagar, PhD, discovered that tiny bits of RNA, called microRNAs, play an important role in Rett, a rare genetic disorder that can impair children’s ability to speak, move and even breathe.

A study by Dr. Zoghbi provides experimental evidence that supports the use of antisense oligonucleotides as a feasible strategy to treat MECP2 Duplication Syndrome. Funding for this work came from the MECP2 Duplication Syndrome Fund at RSRT.

Four girls with Rett Syndrome and their moms discuss what they are grateful for in the "For Goods" section of "Full Circle"

Altering a protein linked to Rett syndrome so that it cannot bind to a particular type of DNA tag may explain the syndrome’s delayed onset in people.

Taysha Gene Therapies provides an update to the Rett Syndrome community on their Rett program, TSHA-102.

The long wait for vaccination has left many people across the country feeling anxious, desperate even. But for the young and disabled, and for the people who care about them, the realization of where they fall on priority lists has also left them feeling disregarded and discarded.

Global Genes interviews Monica Coenraads as part of their Rare Disease efforts.

Taysha Gene Therapies, Inc. has highlighted its strategic priorities and provided a business outlook for 2021.

Neurogene Inc. has announced a collaboration to advance development of multiple platform approaches designed to enable next generation gene therapies.

Two unpublished studies detail improved methods for delivering gene therapies to the brain: One involves a type of stem cell that can produce gene-altering proteins on-site; the other taps an engineered virus to target neurons efficiently and noninvasively.

Clusters of living brain cells are teaching scientists about diseases like autism. With a new finding, some experts wonder if these organoids may become too much like the real thing.