Trehalose Designated Orphan Drug for ALS
SLS-005 contains the active ingredient trehalose, a low molecular weight disaccharide that crosses the blood brain barrier, stabilizes proteins and activates autophagy.
SLS-005 contains the active ingredient trehalose, a low molecular weight disaccharide that crosses the blood brain barrier, stabilizes proteins and activates autophagy.
Median survival 6.5 months longer with initiation of PB-TURSO treatment compared with placebo.
Slower functional decline seen with active drug versus placebo through 24 weeks
The FDA has granted Fast Track designation to arimoclomol (Orphazyme A/S) for the treatment of amyotrophic lateral sclerosis.
Mallinckrodt has permanently discontinued the phase 2b PENNANT trial investigating Acthar Gel (repository corticotrophin) for the treatment of amyotrophic lateral sclerosis (ALS). The study’s independent Data and Safety Monitoring Board (DSMB) recommended the trial be halted after observing a higher rate of pneumonia occurrence in Acthar-treated patients compared with those who received placebo. In addition,…
The FDA approval was supported by data from bioavailability studies comparing riluzole tablets and Tiglutik oral suspension.
Radicava is supplied as a 30mg/100mL solution for intravenous infusion in single-dose polypropylene bags.
Occupations associated with higher education and income show increased mortality risk