The data will be presented at the Annual ALS Drug Development Summit, which is focused on identifying transformative ALS targets, seeking translational biomarkers and propelling more clinical.
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Under a recent law, the National Institutes of Health will begin spending about $25 million to enroll patients in compassionate use – also called open access – programs of unapproved drugs.
Only patients who can t get into conventional drug trials are eligible for the program. And their progress must be tracked to gather data about the treatment and their underlying disease, amyotrophic lateral sclerosis, or ALS.