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Patient case reports

Pyridostigmine

November 12, 2025

Pyridostigmine has a plausible mechanism for transiently enhancing neuromuscular transmission in early stage ALS, where NMJ transmission is impaired but motor neuron numbers are preserved. However, there is no plausible mechanism for slowing ALS progression. Case reports showed PALS without typical MG symptoms did not respond to pyridostigmine. Thus far, clinical trials of pyridostigmine in […]

Glucocorticoid Corticosteroids

August 29, 2022

In conclusion, corticosteroids are a class of medications with wide ranging clinical uses and well studied effects on the immune system. ALS progression is associated with changes in immune system function, with the early disease states associated with anti inflammatory immune markers and the advanced disease states associated withpro inflammatory immune markers. Although treatment with […]

Vitamin C

July 14, 2021

Vitamin C is safe and inexpensive. As an antioxidant, it has a plausible mechanism for influencing the course of neurodegenerative diseases. Two flawed preclinical studies by the same group showed benefits in a mouse model of familial ALS. There are two case reports in which it was associated with improvement. However, there are multiple possible […]

Melatonin

June 2, 2021

Melatonin has plausible mechanisms, some positive (and some negative) pre-clinical data, and two case reports in which it was part of a cocktail of treatments associated with recovery of lost motor function. As we have stated previously, there aremultiple possible explanations for cases like these. There was also a very small, flawed retrospective study suggesting […]

LEAP2BFIT

July 29, 2020

Many ingredients contained within LEAP2BFIT could, at least in theory, be beneficial in ALS. Some of these ingredients have supporting animal or human studies. However, it is unknown if these ingredients are being provided in therapeutic quantities since the dosages are not disclosed. Furthermore, it is impossible to know the net positive or negative effect […]

Curcumin

March 1, 2018

Oral curcumin is safe, inexpensive, and has at least four potential mechanisms by which it might theoretically be useful in treating PALS. Flawed preclinical studies showed benefits of a curcumin chemical analog in a cell model of ALS, three PALS experienced validated motor improvements on regimens including curcumin (although there are several alternative explanations for […]

L-Serine

November 29, 2016

L-serine is a reasonably inexpensive, widely available nutritional supplement that has a plausible mech-anism by which it could help a subset of patients who might have ALS from BMAA-toxicity. A small Phase I trial showed that L-serine up to 15 g twice daily is relatively well tolerated. A larger follow up trial is planned and […]

TUDCA

June 27, 2014

Ursodiol has interesting mechanisms of action, appears reasonably safe and well-tolerated, has anecdotal reports of benefit in 6/21 of patients who report taking it, and a form of it (Yoo’s solution) was associated with slightly slower ALS progression in one out of three outcome measures within a poorly designed study that did not account for […]

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