Amyotrophic lateral sclerosis (ALS) was first described 134 years ago. While still incurable, sig... more Amyotrophic lateral sclerosis (ALS) was first described 134 years ago. While still incurable, significant progress has been made in understanding the pathophysi- ology of the disease and its management. For example, it is now clear that ALS is not a single disease; there are familial and sporadic subtypes. ALS is not specific for motor neurons; other cell types are involved
Tremendous advances in our understanding of pathogenesis of amyotrophic lateral sclerosis (ALS) h... more Tremendous advances in our understanding of pathogenesis of amyotrophic lateral sclerosis (ALS) have provided a rich pipeline of drugs for clinical trialists. At least 32 unique compounds have been tested. Nevertheless, riluzole is currently the only treatment that prolongs survival. We present a critical overview of past clinical trials, how therapies are selected for testing in people, challenges with ALS clinical trial design and conduct, and ways to best move forward.
Clinical trials of potential treatments for amyotrophic lateral sclerosis (ALS) are hindered by t... more Clinical trials of potential treatments for amyotrophic lateral sclerosis (ALS) are hindered by the relative rarity of the disease and the large number of drugs to test. If we tested each drug in a trial of 6001000 patients, in 10 years we would have tested only two or three drugs. Our ...
Amyotrophic lateral sclerosis (ALS) was first described 134 years ago. While still incurable, sig... more Amyotrophic lateral sclerosis (ALS) was first described 134 years ago. While still incurable, significant progress has been made in understanding the pathophysi- ology of the disease and its management. For example, it is now clear that ALS is not a single disease; there are familial and sporadic subtypes. ALS is not specific for motor neurons; other cell types are involved
Tremendous advances in our understanding of pathogenesis of amyotrophic lateral sclerosis (ALS) h... more Tremendous advances in our understanding of pathogenesis of amyotrophic lateral sclerosis (ALS) have provided a rich pipeline of drugs for clinical trialists. At least 32 unique compounds have been tested. Nevertheless, riluzole is currently the only treatment that prolongs survival. We present a critical overview of past clinical trials, how therapies are selected for testing in people, challenges with ALS clinical trial design and conduct, and ways to best move forward.
Clinical trials of potential treatments for amyotrophic lateral sclerosis (ALS) are hindered by t... more Clinical trials of potential treatments for amyotrophic lateral sclerosis (ALS) are hindered by the relative rarity of the disease and the large number of drugs to test. If we tested each drug in a trial of 6001000 patients, in 10 years we would have tested only two or three drugs. Our ...
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