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Rett In The News

Increasing expression of the gene that is faulty in Rett syndrome lowers repetitive behaviors in a mouse model of a different autism-linked condition called Pitt-Hopkins syndrome, a new unpublished study demonstrates.
Taysha Gene Therapies
Taysha Gene Therapies today announced that it has been granted orphan drug designation from the European Commission for TSHA-102, an AAV9-based gene replacement therapy in development for Rett syndrome. An orphan designation allows a pharmaceutical company to benefit from incentives such as ...
A new class of therapies based on transfer RNA could treat forms of cystic fibrosis, muscular dystrophy, genetic epilepsies, and more
UCLA researchers identify an experimental drug that improves abnormal neural oscillations found in Rett brain organoids.
Taysha Gene Therapies
Taysha Gene Therapies announces ambitious timeline of starting Rett syndrome gene replacement clinical trial by year end and reporting clinical data by end of 2022.
The Undefeated
Olympic bronze medal winner Gabby Thomas studied neurobiology at Harvard and worked on Rett syndrome in a lab at Boston Children’s Hospital.
Shape Therapeutics
Shape Therapeutics, a biotech focusing on RNA editing, has secured $112 million in financing to advance their research programs. They have two Rett Syndrome programs one of which is their lead program. We’re proud that RSRT has facilitated Shape taking on Rett.
Endpoints News
Two years after spinning out of CRISPR pioneer Prashant Mali’s lab, Shape Therapeutics has a lot more cash and a slightly new mission. They have lead programs in Rett syndrome and Parkinson’s.
Scientists in France published a paper showing that the MECP2 protein binds certain repeating sequences in strands of DNA thereby changing its structure. Mutated MECP2 could not bind and therefore no change in DNA strucuture was seen. If and how this finding is relevant to developing treatments ...
Fierce Biotech
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 ...
St. Thomas Source
Ella Gaspard, a Virgin Islander, is diagnosed with Rett Syndrome. Ella’s Hope invites the public to join it on the first STT Cross-Island Challenge Ride to be held on July 3. One hundred percent of all proceeds raised from the STT Cross-Island Challenge will be donated to Rett Syndrome ...
Endpoints News
In downtown Lowell, MA, tucked into one of the country’s oldest surviving textile mill complexes is a new biotech toiling away on next-gen CNS therapies for severe neurological disorders. And it’s starting with Rett syndrome — a rare neurodevelopmental condition that almost exclusively ...
Endpoints News
“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.
Endpoints News
RNA used to be considered a bit player. DNA was the start. Today it's a different story....
Shoutout LA
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
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.
Behavioral Training Could Help Babies with Rett Syndrome, Mouse Study Suggests
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.
University of Connecticut
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 Health
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.
Texas Children's Hospital
ASO as a feasible therapy to treat MECP2 duplication disorder
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.
Rett Syndrome Awareness on Anderson Coooper's Full Circle
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.