Steve Bryson, PhD, science writer —

Steve holds a PhD in biochemistry from the Faculty of Medicine at the University of Toronto, Canada. As a medical scientist for 18 years, he worked in both academia and industry, where his research focused on the discovery of new vaccines and medicines to treat inflammatory disorders and infectious diseases. Steve is a published author in multiple peer-reviewed scientific journals and a patented inventor.

Articles by Steve Bryson

Enzyme replacement therapy found effective in Sanfilippo type A mice

The investigational, brain-penetrating enzyme replacement therapy DNL126 was found to correct neurological signs of Sanfilippo syndrome type A in a mouse model, according to its developer, Denali Therapeutics. Now, the biopharmaceutical company plans to seek U.S. Food and Drug Administration permission — via an investigational new drug application…

Metabolites in patients’ urine may be markers of disease, ERT response

Researchers identified 11 new candidate metabolite biomarkers in the urine of people with Sanfilippo syndrome, a study reported. Metabolomics, or examining global changes in metabolites in metabolic disorders such as Sanfilippo syndrome, may shed light on underlying disease mechanisms and provide a source of noninvasive biomarkers to support diagnosis,…

Increased Disease Severity Found With Age in Sanfilippo Youth

Neurological, behavioral, and MRI examinations of a group of children and adolescents with Sanfilippo syndrome in Brazil showed increased disease severity with age that coincided with progressive brain involvement, according to a new study. Changes in all areas increased in prevalence as the children grew, the researchers noted in…

Parents, Caregivers Vital in Sanfilippo Disease Research: Study

According to parents of children with Sanfilippo syndrome, developmental delays, hyperactivity, sleep disturbances, hearing loss, and recurrent infections were the most common features of the condition. This study adds to the natural history of Sanfilippo syndrome and underscores the vital role parents and caregivers play in disease research, the…

Impaired Energy Balance May Explain Sanfilippo Energy Demands

A mouse model of Sanfilippo syndrome had persistent energy demands driven by activated heat-generating fat cells and impaired recycling of energy-producing mitochondria, a study revealed. These findings may explain the negative energy balance seen in Sanfilippo patients, which can lead to wasting in severe cases, despite adequate food intake,…

Gene Therapy LYS-SAF302 Boosts or Stabilizes Development After 2 Years

Children with Sanfilippo syndrome type A, including those with severe disease, showed stable or continuously increasing cognitive, language, and motor functions two years after dosing with the investigational gene therapy LYS-SAF302, new data from the AAVance trial showed. The therapy also maintained low levels of the disease-related biomarker heparan sulfate…

Changes Seen in Brains of Sanfilippo D Mice May Advance Therapy Work

An examination of brain tissue from a mouse model of Sanfilippo syndrome type D revealed several progressive disease-related features, including the altered degradation of energy-producing mitochondria and the abnormal activation of immune cells associated with inflammation and brain cell death, a study reported. Findings show key underlying processes associated…