Target Health Blog

Lysosomal Storage Diseases (LSDs) Conference

November 18, 2019

What's New

In Lysosomal Storage Diseases (LSDs), neurological complications are quite common. Advanced therapies for LSDs that have been in pipeline include gene therapy, small molecule/nanomedicine approaches, and genome editing. Although newer approaches seem promising, there is no consensus on outcome measures or specific biomarkers to define the neurological involvement in LSDs.

Target Health, LLC. Is pleased to announce that Dr. Jules Mitchel, CEO, will participate as a speaker in a workshop on "Neurological Outcomes in Lysosomal Storage Disorders" organized by Lysosomal & Rare Disorders Research & Treatment Center (LDRTC). The Genetic, Rare Immune Disorders Symposium (GRIDS) workshop will take place in Fair Oaks Marriott Hotel, Fairfax, Virginia on November 24-25, 2019. The presentation is entitled “Contract research organizations in conducting clinical trials in neurological disorders.“ The workshop will focus on basic mechanisms behind the pathophysiology and establishing clinical outcome measures for clinical trials and disease monitoring. The program will also include discussions on emerging treatment strategies as well as the process of clinical trial development for neuropathic LSDs.

Here is some background information: Nervous system poses a major challenge not only to understand the primary pathology of a given disease state, but manage patients clinically, and conduct clinical trials for drug development. Less than 10% of newly developed drugs on the market target a primary nervous system disorder. For example, within the past 10 years, while more than 50% of cancer therapeutics in trials received an FDA approval, all drugs targeting Alzheimer disease, virtually have failed. Multiple factors, including incorrect therapeutic targets, imprecise, misleading or inaccurate clinical methodologies, and the delay from the onset of actual pathologic process to the clinical recognition of symptoms could be listed as some of reasons for the lack of success in medical field to tackle neurological disorders.

For more information about Target Health, contact Warren Pearlson (212-681-2100 ext. 165). For additional information about software tools for paperless clinical trials, please also feel free to contact Dr. Jules T. Mitchel. The Target Health software tools are designed to partner with both CROs and Sponsors. Also visit the Target Health Eating Website to see all of the fantastic recipes since 2012.

Joyce Hays, Founder and Editor in Chief of On Target

Dr. Jules T. Mitchel, Editor

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