A medication that’s currently used to treat Parkinson’s disease showed potential for addressing breathing problems associated with Rett syndrome in a mouse study. Scientists found that abnormal activity in the carotid body, a small structure in the neck that helps monitor oxygen levels in the blood, contributed to Rett-like…
News
Blocking HSF1, a protein active during the cellular stress response, may be a novel therapeutic target for easing symptoms in Rett syndrome, a new study suggests. Researchers found that an overactive cellular stress response driven by HSF1 appeared to worsen symptoms in Rett mice. Blocking the HSF1 pathway —…
The European Commission has approved Acadia Pharmaceuticals’ Daybu (trofinetide) — sold elsewhere as Daybue — as the first treatment authorized in the European Union for disease-related behavioral, emotional, and communication issues in people with Rett syndrome, ages 5 and older. The oral therapy, taken by mouth or via a…
With a Phase 3 trial currently in progress, Neurogene is getting ready to file an application seeking approval of NGN-401, the company’s experimental gene therapy for Rett syndrome. Neurogene recently completed dosing in the Phase 3 Embolden clinical trial (NCT05898620), which is testing NGN-401 in 25…
Shape Therapeutics and the Rett Syndrome Research Trust (RSRT) are teaming up to advance the development of SHP-401, Shape’s experimental one-time gene therapy for certain people with Rett syndrome. RSRT will fund tests of SHP-401 in non-human primates to evaluate the therapy’s distribution in the body and…
Taysha Gene Therapies is preparing for the potential commercial launch of TSHA-102, its experimental gene therapy for Rett syndrome now in pivotal clinical testing. To support the potential launch, Taysha recently announced a commercial supply agreement with Catalent, a pharmaceutical manufacturing company. Under the agreement, Catalent will be…
Modifications to MeCP2, the protein whose dysfunction underlies most cases of Rett syndrome, may help explain the wide range of symptoms seen in people with the condition, a new mouse study suggests. The study found that combining two chemical MeCP2 modifications produced complex behavioral and molecular effects that differed…
Only a small percentage of boys with Rett syndrome received treatment with Daybue (trofinetide) in the more than one year after the therapy was approved in the U.S., according to a real-world study of healthcare claims data. Before treatment, boys treated with Daybue had a greater burden of…
Stimulating the vagus nerve — a large nerve that helps control unconscious bodily functions — partly normalized some measures of sound-related brain activity in a rat model of Rett syndrome, a new study reports. Although vagus nerve stimulation, or VNS, improved some measures of sound-related brain activity, including how…
All 10 patients with Rett syndrome treated with Neurogene‘s gene therapy NGN-401 continued to make developmental gains over 2.5 years of follow-up, with no plateau or loss of skills observed, according to long-term data from a Phase 1/2 clinical trial. The data showed that these gains deepened over time…
Recent Posts
- Feeling adrift following the end of Camp Communicate
- Parkinson’s drug shows potential for Rett breathing problems in mice
- A casual chat about Rett syndrome, then a surprise in the mail
- Blocking cellular stress protein may help ease Rett symptoms
- My daughter with Rett syndrome will never ride Medusa — and neither will I
- Learning to view mobility aids and other medical equipment as vital tools
- Daybu wins approval in EU as region’s first Rett syndrome treatment
- Rett syndrome as a windowless warehouse, and my daughter inside
- Neurogene gearing up to apply for approval of Rett gene therapy
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