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Positive Results from Two Phase 3 Trials for Potential New Treatment for Fabry Disease

Positive Results from Two Phase 3 Trials for Potential New Treatment for Fabry Disease

Fabry Disease Fabry Disease is a rare lysosomal storage condition. Mutations in the GLA gene cause a type of fat called globotriaosylceramide to build up in the body's cells. Symptoms…

Continue Reading Positive Results from Two Phase 3 Trials for Potential New Treatment for Fabry Disease
A Failed Cancer Drug Shows Promise Treating Animal Models of Dementia

A Failed Cancer Drug Shows Promise Treating Animal Models of Dementia

According to a publication from Medical News Today, in animal studies, a failed cancer drug showed signs of promise in treating frontotemporal dementia. Frontotemporal Dementia Dementia is a blanket term…

Continue Reading A Failed Cancer Drug Shows Promise Treating Animal Models of Dementia

Updates in Experimental Therapies for Liver Disease

According to a story from Healio, there are a number of recent reports of progress in the development of treatments for rare forms of liver disease. Many rare liver diseases…

Continue Reading Updates in Experimental Therapies for Liver Disease
Formal Appeal to the CDC to Revise Incorrect Data Published on Lyme Disease

Formal Appeal to the CDC to Revise Incorrect Data Published on Lyme Disease

Bruce Alan Fries, President of the Patient Centered Care Advocacy Group, along with Holly Ahern and Chris Fisk from the Lyme Action Network, have issued an official "Information Quality Request…

Continue Reading Formal Appeal to the CDC to Revise Incorrect Data Published on Lyme Disease
Proof of Concept Results Achieved in Congenital Adrenal Hyperplasia Trial

Proof of Concept Results Achieved in Congenital Adrenal Hyperplasia Trial

According to a story from BioSpace, the biotechnology company Spruce Biosciences recently announced that they have achieved positive proof of concept data in the company's clinical trial. This is a…

Continue Reading Proof of Concept Results Achieved in Congenital Adrenal Hyperplasia Trial
Preventive Use of Extended Half-Life Therapies Provides Enhanced Benefit for Hemophilia A Patients

Preventive Use of Extended Half-Life Therapies Provides Enhanced Benefit for Hemophilia A Patients

According to a story from BioSpace, a recent study revealed that hemophilia A patients were less likely to experience bleeds when they used new, extended half-life therapies on a preventive…

Continue Reading Preventive Use of Extended Half-Life Therapies Provides Enhanced Benefit for Hemophilia A Patients
Data From Early Short Bowel Syndrome Trials Presented

Data From Early Short Bowel Syndrome Trials Presented

According to a story from PR Newswire, the biopharmaceutical company Therachon AG recently issued a presentation of results from the company's Phase I clinical trial. This clinical trial tested the…

Continue Reading Data From Early Short Bowel Syndrome Trials Presented
Gene Therapy Proves Effective in Treating AADC Deficiency in Trial

Gene Therapy Proves Effective in Treating AADC Deficiency in Trial

According to the publication over the weekend from San Francisco CBS affiliate KPIX, a groundbreaking clinical trial for those with aromatic l-amino acid decadboxylase (AADC) deficiency was undertaken at UCSF…

Continue Reading Gene Therapy Proves Effective in Treating AADC Deficiency in Trial
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