Preclinical Findings for Phenylketonuria and Metachromatic Leukodystrophy Gene Therapies Look Promising

According to a story from BioSpace, the genetic medicines company Homology Medicines, Inc., has recently released preclinical data supporting the development of two experimental gene therapies. One, called HMI-202, is…

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The First Clinical Evidence that the Drug Mavoglurant Improves Responsiveness in Fragile X Syndrome (FXS)

Results of a study reported in PLOS ONE that was conducted by a team of scientists indicate that the experimental drug mavoglurant improves responsiveness and eye gaze for patients with…

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First Patient to Receive Gene Therapy in a Phase 1/2 Study of ACTUS-101 in Patients with Pompe Disease

Actus Therapeutics, a privately held portfolio company of AskBio, has recently announced through PRWeb, the initiation of patient dosing in a clinical trial testing its investigational gene therapy ACTUS-101. About Pompe…

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Healx Ltd. Raises $56 Million to Use Artificial Intelligence to Sped Up Rare Disease Drug Trials
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Healx Ltd. Raises $56 Million to Use Artificial Intelligence to Sped Up Rare Disease Drug Trials

As originally reported in South Africa Latest News, Healx Ltd. has raised $56 million dollars to fund their development of treatment options for rare diseases using artificial intelligence. The British…

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