Seven-year-old Rydder Ames of Hamilton, Ohio began having troubling symptoms five months ago. His mother, Krista Steven took him to the ER after a week-long headache followed by a…
According to a press release, the gene therapy company bluebird bio, inc. has recently announced that its gene therapy elivaldogene autotemcel (marketed as SKYSONA) has received Accelerated Approval status from…
FBXL4-related mitochondrial disease urgently requires new therapies. Unfortunately, this condition is often fatal by early ages (early to mid-childhood). Therefore, it is important to spur research into potential interventions or…
Currently, an estimated 187,000 children within the United Kingdom, and millions worldwide, have some sort of arm disability. This can be linked to damage or various conditions, such as cerebral…
In the United States, the FDA grants Orphan Drug designation to drugs or biologics intended to prevent, diagnose, or treat rare conditions. These are conditions affecting fewer than 200,000 Americans.…
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