News

Mental Rigidity in PWS Goes Beyond Intellectual Disability

Adults with Prader-Willi syndrome (PWS) have greater mental rigidity than those with matching intellectual disability, but without the disease, and those with normal intellectual level, according to a study from France. These findings support the presence of PWS-specific mental flexibility deficits, over and above intellectual problems, the researchers said.

Soleno Looking to Expand Potential Therapies That Work Like DCCR

Soleno Therapeutics has partnered with Vanderbilt University to develop therapies that work as ATP-dependent potassium channel activators for rare diseases, similar to the diazoxide choline controlled release (DCCR) tablets the company thinks might treat Prader-Willi syndrome (PWS). ATP-dependent potassium (or KATP) channels are proteins present in multiple body tissues.

Rare Disease Day at NIH, Set for March 1, Growing Year by Year

Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, research updates, TED-style talks, and a presentation by a Nobel laureate recently recognized for her work on a gene editing tool. The free, virtual…

Tonix Licenses Via-the-nose Treatment Technology From Inserm

Tonix Pharmaceuticals has licensed technology from the French National Institute of Health and Medical Research (Inserm) that it aims to use for treating failure to thrive in Prader-Willi syndrome. The license will allow Tonix to expand its intranasal — administered via the nose — potentiated oxytocin program to…