Author Archives: Steve Bryson PhD

Experimental Rett Therapy Now an Orphan Drug in Europe

The European Commission has granted orphan drug designation to TSHA-102, an investigational gene therapy for the treatment of Rett syndrome. The designation encourages the development of medicines to diagnose, prevent, or treat life-threatening or debilitating diseases that affect fewer than five in 10,000 people living in the European…

Comparative Genomics Identifies 3 Potential Therapies

Using a comparative genomics approach, researchers have identified three potential therapies for Rett syndrome that are being used or tested for other indications. “This study highlights the potential of comparative genomics to accelerate drug discovery, and yields potential new avenues for the treatment of [Rett],” the researchers wrote. …

3D Brain Organoids Useful as Rett Syndrome Model, Study Shows 

Brain organoids grown from stem cells derived from people with Rett syndrome reproduced patterns of electrical brain activity that resembled seizures, a hallmark of the condition, a study demonstrated. Use of anti-seizure medications restored electrical activity to normal levels, supporting the use of brain organoid models to investigate underlying…

Daytime Teeth Grinding Means More Dental Work

People with Rett syndrome who grind their teeth during the day had a higher incidence of dental treatment, particularly restorations, than those who did not, an international database study reported.  The findings also showed that patients fed orally had more dental…