AVROBIO Provides Updates on its Fabry Disease Program and Cystinosis Program
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AVROBIO Provides Updates on its Fabry Disease Program and Cystinosis Program

According to a story from Financial Buzz, the gene therapy company AVROBIO recently presented updated data on February 10th, 2020 at the 16th Annual WORLDSymposium being held in Orlando, FL. These updates…

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Combination Treatment for Myelofibrosis Shows Potential in Early Trial
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Combination Treatment for Myelofibrosis Shows Potential in Early Trial

According to a story from Targeted Oncology, findings from a recent phase 1 trial have discovered a new potential treatment for myelofibrosis. The study combined two different drugs: panobinostat (marketed…

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Researchers Have Found New Causes of Angiosarcoma and 3 Trials are Underway
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Researchers Have Found New Causes of Angiosarcoma and 3 Trials are Underway

Angiosarcoma Angiosarcoma is a rare condition caused by a tumor within the endothelial cells. These cells line the blood vessels in the body, and therefore the tumors can occur practically…

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Neurogene and Behind the Seizure Work to Provide Better Diagnostics for Batten Disease
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Neurogene and Behind the Seizure Work to Provide Better Diagnostics for Batten Disease

Neurogene, which works to provide treatments for rare neurological disorders, has recently joined with Behind the Seizure. Behind the Seizure was established by two other companies, BioMarin and Invitae, with…

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First Patient Enrolled in a New Methylmalonic Acidemia Trial
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First Patient Enrolled in a New Methylmalonic Acidemia Trial

Methylmalonic Acidemia Methylmalonic acidemia (MMA) is a rare disease affecting the metabolic system. 60% of all cases are due to a deficiency in MUT, a mitochondrial enzyme, which causes acids to…

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