Glycogen Storage Disease Clinical Trials 2023

What are the top hospitals conducting glycogen storage disease research?

In the realm of medical research, hospitals across the United States are making significant strides in understanding and treating glycogen storage disease. Among them, the University of Connecticut Health Center in Farmington has emerged as a pioneer with two active clinical trials dedicated to this rare condition. While their overall contribution may seem modest at this point, it is worth noting that they only recently recorded their first glycogen storage disease trial in 2021.

Meanwhile, the University of Utah in Salt Lake City follows closely behind with two ongoing trials of their own. With an equally impressive history of two all-time trials since stepping into this field back in 2020. The hospital's dedication to unraveling the complexities of glycogen storage disease showcases its commitment to improving patient outcomes.

The Children's Hospital at Montefiore in Bronx recognizes the significance of addressing glycogen storage disease by conducting one currently active clinical trial on top it being partaken over years for respective studies concerning such conditions where they have had initiated one study earlier previous year before proceeding further onto next levels from 2021.

At Philadelphia’s renowned Children's Hospital, researchers are working diligently on a single active clinical trial aimed at better understanding and managing glycogen storage disease; however, they have previously conducted three other such investigations within this specialized area since recording their first trial also around early months during similar time frame just like these others aforementioned hospitals did so too throughout 2021

Lastly but not leastly ,the University of Texas Health Science Center located within Houston adds itself unto prestigious list featuring available resources nationwide for people suffering from Glycogen Storage Disorders ;having run number series upto another ongoing initiative whilst keeping record total up until now reaching out involving two experiments spearheading towards working knowledge ever more about any similar cases including updated results which were initially begun way back much prior nearly four years ago situated sometime around2019

These distinguished institutions' efforts collectively serve as beacons hope patients affected by Glycogen Storage Diseases, as well as a testament to the relentless pursuit of medical progress. While the number of trials might appear small in comparison to other conditions, these hospitals are laying the foundation for further advancements and bringing us one step closer to improved treatments and quality of life for those with glycogen storage disease.

Which are the best cities for glycogen storage disease clinical trials?

Glycogen storage disease clinical trials are thriving in several cities across North America. Durham, North carolina emerges as a top contender with 5 active trials investigating promising treatments such as DTX401, mRNA-3745, and Avalglucosidase alfa. Houston, Texas follows closely behind with 4 ongoing studies exploring DTX401 and mRNA-3745 among others. Salt Lake City, Utah and Vancouver, British Columbia both have 3 active trials focusing on DTX401 and other potential breakthroughs like SPK-3006 and carbohydrates intake respectively. Lastly, Farmington, Connecticut contributes to the research efforts with 2 active trials primarily centered around DTX401 and mRNA-3745. These cities offer individuals affected by glycogen storage diseases access to cutting-edge clinical trials that pave the way for advancements in care and treatment options.

Which are the top treatments for glycogen storage disease being explored in clinical trials?

Glycogen storage disease is a complex condition, but clinical trials offer hope for new treatments. Among the top contenders are:

• DTX401: Currently in 1 active trial and has been part of 2 all-time glycogen storage disease trials since its listing in 2018.
• mRNA-3745: Showing promise, this treatment is being explored in 1 ongoing trial and marks the first appearance on the scene in 2022.
• SPK-3006: Another potential breakthrough, this therapy is currently undergoing one active trial and made its debut in clinical research back in 2020. With these innovative treatments under investigation, there's renewed optimism for those affected by glycogen storage disease.

What are the most recent clinical trials for glycogen storage disease?

Recent advancements in clinical trials offer hope for individuals with glycogen storage disease. Promising studies include the investigation of Cipaglucosidase Alfa/Miglustat in pediatric patients who have previously undergone enzyme replacement therapy for infantile-onset Pompe disease, as well as the evaluation of mRNA-3745 and DTX401 treatments for glycogen storage disease. Another notable trial focuses on Avalglucosidase alfa, which aims to improve treatment outcomes. Furthermore, SPK-3006 is being studied in both Phase 1 and Phase 2 trials to explore its potential benefits. These groundbreaking trials highlight the ongoing efforts to develop effective therapeutic options and enhance quality of life for those affected by glycogen storage disease.

What glycogen storage disease clinical trials were recently completed?

A recent clinical trial has provided valuable insights in the treatment of glycogen storage disease. Sponsored by Genzyme, a Sanofi Company, the trial examined Avalglucosidase alfa (GZ402666). Concluded in November 2016, this study contributes to our understanding of potential therapeutic options for individuals affected by this metabolic disorder. Although further research is necessary, these findings offer hope for improved management and outcomes in patients with glycogen storage disease.