New Muscle Therapy Gets Fast-Track Boost

To help bring therapies for rare muscle diseases in children to market sooner, the Berlin-based start-up MyoPax, a spin-off from the Max Delbrück Center and Charité – Universitätsmedizin Berlin, has now received a boost from the U.S. Food and Drug Administration (FDA). The company has been granted the FDA’s orphan drug designation (ODD) and rare pediatric disease designation (RPDD), both of which offer multiple regulatory and financial advantages – including fast-track approval status and, eventually, market exclusivity. Read more.

Source: Eurekalert, July 26, 2023

High Dry Night Rate for Users of New Bedwetting AI Alarm

High compliance users of the new bedwetting alarm, GOGOband, which utilizes real-time heart rate variability analysis and applies artificial intelligence (AI) to create an alarm to wake the user, have a 93 percent dry night rate, according to a study published online April 28 in the Journal of Pediatric Urology. Israel Franco, M.D., from the Yale University School of Medicine in New Haven, Connecticut, and Jon Coble, from GoGoband Inc. in Richmond, Virginia, evaluated a new bedwetting alarm, GOGOband, and assessed the efficacy in the first 18 months of use. All 54 individuals (mean age, 10.1 years) who used the system for more than 30 nights from Jan. 1, 2020, to June 2021, were included. Read more.

Source: Health Day, May 30, 2023

Bristol-based children’s incontinence charity marks its 30th anniversary

The UK’s only charity dedicated to bowel and bladder health in children and teenagers in the UK – based in Bristol – today (May 1) marks its 30th anniversary. ERIC has been providing valuable support and advice to parents and children struggling with incontinence, a condition that continues to be heavily stigmatised, for more than three decades and has commemorated its big birthday by making the help it provides even more accessible. In the UK an estimated 900,000, or 1 in 12, 5-19 year olds suffer from bowel and bladder conditions – including bedwetting, daytime wetting, constipation and soiling. Read more.

Source: BristolLive, May 1, 2019

Charting an Underexplored Landscape: The Genitourinary Microbiome

More sensitive cultivation methods and precise 16S rRNA gene sequencing techniques have revealed that the human bladder hosts a significant microbiome and those diverse bacteria inside the bladder impact pediatric urologic diseases. As recently as one decade ago, the human bladder was thought to be a sterile landscape. In recent years that view has shifted radically, opening brand-new fields of research aimed at clarifying the role the microbiome plays in common urologic diseases that affect children, according to a review article published online Feb. 22, 2018, by Current Urology Reports. “There is a growing appreciation for the role of diverse bacteria in contributing to improved health as well as triggering disease processes or exacerbating illness,” says Michael H. Hsieh, M.D., Ph.D., director of the Clinic for Adolescent and Adult Pediatric Onset Urology (CAPITUL) at Children’s National Health System and study senior author. Read more.

Source: Medical Xpress, APril 5, 2018