Advanced Treatment of Amyotrophic Lateral Sclerosis (ALS)

Jamal Bennour, Mohammed (2019-11-21)

Amyotrophic lateral sclerosis (ALS) is the most common form of the neurodegeneration , is progressive and fatal neuromuscular disease , the disease characterized by the degeneration of motor neurons in the brain and spinal cord , when the motor neurons die , the ability of the brain to control muscle movement lost, the people loss the ability to speak , move , eat , breath . Approximately half of (ALS) patients die within 3-5 years of symptoms onset , the (ALS) usually strikes people between the age 40-70 , the disease is discovered by French doctor named ( jean-martin charcot) , “ Amyotrophy “ refers to the atrophy of the muscle fibers , which are denervated as their corresponding anterior horn cell degenerate , “ Lateral sclerosis” refers to hardening of the anterior and lateral corticospinal tracts as motor neurons in these areas degenerate and are replaced by gliosis .


Background: Amyotrophic lateral sclerosis (ALS) is a progressive and fatal neuromuscular disease that affects approximately 5 of every 100,000 individuals, Approximately half of ALS patients die within 30 mo of symptom onset and the majority do not survive beyond 5 years, Currently, riluzole and edaravone are the only United States Food and Drug Administration (FDA)–approved treatment options Methods: Outcome (the change in ALS Functional Rating Scale–Revised, ALSFRSR, from baseline) was projected for placebo patients through 48 weeks and compared with 48-week edaravone or 24-week edaravone after switching from placebo. Results: A total of 123 patients received open-label treatment (65 edaravoneedaravone; 58 placebo-edaravone). The projected ALSFRS-R decline for placebo from baseline through week 48 was greater than for 48-week edaravone (P < .0001). For patients switching from placebo to edaravone, ALSFRS-R slope approached that of continued edaravone for 48 weeks. ALSFRS-R decline did not differ between actual and projected edaravone through week 48. Conclusions: Compared with placebo, these analyses suggest that edaravone is beneficial in ALS patients even after 6 mo of receiving placebo, and efficacy is maintained for up to 1 year.

Attribution 3.0 United States
Except where otherwise noted, this item's license is described as Attribution 3.0 United States