Rett In The News

The Massachusetts biotech unveiled itself Wednesday with $23 million in funding from RTW Investments with a pipeline of 12 adeno-associated virus (AAV) gene therapy programs for severe CNS disorders. Alcyone lead programs include ACTX-101, a Rett syndrome gene therapy in pre-IND enabling ...

“This is the way to do it, to discover drugs for neurological diseases,” says Sharath Hegde “And it’s not going to be easy, but this is the way to do it.” And the company's lead program is Rett Syndrome.

Herophilus appoints a new Chief Scientific Officer and sets its sight on Rett Syndrome.

RNA used to be considered a bit player. DNA was the start. Today it's a different story....

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.