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NurOwn is the clearest test yet of how far the agency may be willing to bend to approve a new medicine for a rare and deadly condition with few treatment options. ALS gradually destroys nerve ...
Health reporter Anjalee Khemlani outlines the circumstances behind the FDA's approval of a new ALS treatment drug amid high drug prices.
An experimental medication may slow the progression of amyotrophic lateral sclerosis, or ALS, researchers reported Wednesday. The research was supported in part by donations from the Ice Bucket ...
Tofersen, sold under the brand name Qalsody, is a medication used for the treatment of amyotrophic lateral sclerosis (ALS). [2] Tofersen is an antisense oligonucleotide that targets the production of superoxide dismutase 1, an enzyme whose mutant form is commonly associated with amyotrophic lateral sclerosis.
Project ALS is a non-profit organization whose mission is to identify and support leading scientific research toward the first effective treatments and a cure for ALS. Founded in 1998 by Jenifer Estess , Valerie Estess, Meredith Estess, and Julianne Hoffenberg, Project ALS recruits scientists and doctors to work together toward a better ...
FDA leaders have recently emphasized a new level of “regulatory flexibility” when reviewing experimental treatments for fatal, hard-to-treat conditions, including ALS, Alzheimer’s and ...
Familial ALS is the most studied; however, a new technique that was recently introduced is the use of induced pluripotent stem cells (iPSC). [2] In this study the researcher can isolate skin fibroblast from a patient with familial or sporadic ALS and reprogram them into motor neuron to study ALS. [ 2 ]
This marks the latest setback in the long list of roadblocks in the path to develop effective treatments against amyotrophic lateral sclerosis (ALS), a condition which affects 16,000 to 32,000 ...