The treatment, developed at the University of Pennsylvania with clinical trials carried out at Children's Hospital of Philadelphia and the University of Iowa, is the first time gene therapy is used to treat an inherited disease and could lead to treatments of other
currently untreatable conditions.
«This study represents an important step towards developing a potential therapeutic option for this devastating and
currently untreatable condition.
Using this technology some forms of blindness may be treatable, including macular degeneration and Stargardt disease,
a currently untreatable form eye disease that causes blindness in teenagers and young adults.
As a result, Moderna's platform has the potential to speed the development and manufacture of treatments for many diseases that are
currently untreatable with existing pharmaceutical approaches.
And because other foreign molecules can be substituted for the cat protein, the method offers hope for people with
currently untreatable allergies such as those to nuts and seafood.
Researchers have identified the cause of chronic, and
currently untreatable, pain in those with amputations and severe nerve damage, as well as a potential treatment which relies on engineering instead of drugs.
Results of a phase 2 study published in The Lancet suggest that simvastatin, a cheap cholesterol lowering drug, might be a potential treatment option for the secondary progressive, or chronic, stage of multiple sclerosis (MS), which is
currently untreatable.
Australian researchers have made a critical discovery about a gene involved in muscular dystrophy that could lead to future therapies for
the currently untreatable disease.
Some of these people have vision problems caused by
currently untreatable diseases, he notes, but others simply because they can not afford or do not have access to relatively simple fixes such as surgery to remove cataracts (clouding of eye lenses).