I recently had the pleasure of being interviewed by Sunshine Mugrabi about how I transformed my life over the past 20 years in the face of immense pain and suffering…
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…
Hypotonia Ataxia, and Delayed Development Syndrome Hypotonia, Ataxia, and Delayed Development Syndrome (HADDS) is caused by mutations in the EBF3 gene which is found on chromosome 10q26. Its discovery just…
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…
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…
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…
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…
Steffanie Strathdee and her husband Tom Patterson are both scientists who work at the University of California. On an innocent trip to Egypt were they spent a romantic night…
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