According to a story from BioSpace, the biopharmaceutical company Orphazyme recently announced the results from its Phase 2/3 clinical trial of its investigational drug arimoclomol. This therapy is in development…
According to a story from BusinessWire, the drug developer Alexion Pharmaceuticals, Inc., recently announced positive results for its Phase 3 trial testing Soliris as a therapy for neuromyelitis optica spectrum…
The United States Food and Drug Administration has awarded Orphan Drug Designation to Q-Cells®, a product being developed by Q Therapeutics, for the treatment of transverse myelitis. For more detailed…
Avrobio has released updates on patients in two clinical trials of a potential gene therapy for Fabry disease. According to the announcement, two patients taking part in a Phase 1…
Ultragenyx Pharmaceuticals has shared data from two cohorts taking part in an on-going study involving an investigational gene therapy for ornithine transcarbamylase deficiency. For more information, you can view the…
My name is Lindsay Fogarty. I am a 25-year-old single mother to a wonderful 6, almost 7-year-old son, named Andrew. As I am writing this I am listening to the…
Topline results have been shared from a Phase 3 study of the investigational drug givosiran in patients with acute hepatic porphyria. For more detailed information you can view the source…
Many people express skepticism about the pharmaceutical industry. If not skepticism, than concern, if not wholesale distrust. For every few negative stories, however, there is at least one positive example.…
Many persons living with rare disease require a transplant of certain organs. Though this is not a cure for the rare disease Cystinosis, a kidney transplant is often required once the disease…
According to a story from Parkinson's News Today, a recent analysis of Parkinson's disease research showed that scientists would benefit from relying less on animal models and instead should focus…
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