Cellular repair system could prevent protein aggregation in neurodegenerative disease ALS

A small-molecule inhibitor of SOD1-Derlin-1 interaction ameliorates pathology in an ALS mouse model

Amyotrophic lateral sclerosis (ALS) is a devastating neurodegenerative disorder. Despite its severity, there are no effective treatments because of the complexity of its pathogenesis. As one of the underlying mechanisms of Cu, Zn superoxide dismutase (SOD1) gene mutation-induced ALS, SOD1 mutants (SOD1mut) commonly interact with an endoplasmic reticulum-resident membrane protein Derlin-1, triggering motoneuron death. However, the importance of S…

Cellular repair system could prevent protein aggregation in neurodegenerative disease ALS

Amyotrophic lateral sclerosis (ALS) is a relatively rare but very serious disease of the nervous system. A cure is not yet possible. ALS slowly destroys the motor neurons in the brain and spinal cord responsible for voluntary muscle control. The result is progressive muscle paralysis, leaving many patients wheelchair dependent. As the disease progresses, individuals find it increasingly difficult to speak, swallow, and breathe.

SOD1-ALS disease progression may slow with RAG-17: Final trial data

An experimental candidate by Ractigen Therapeutics to treat amyotrophic lateral sclerosis (ALS) linked to mutations in the SOD1 gene has shown promising signs of slowed functional decline and stable or improved breathing in ALS patients of a small early trial. The therapy, RAG-17, also significantly reduced blood levels of neurofilament light chain (NfL), a biomarker of nerve damage, and demonstrated target engagement, as indicated by a drop in …

Global Amyotrophic Lateral Sclerosis Market Innovations Challenges and 7-8% CAGR by 2029 - Daily Investor Hub

The amyotrophic lateral sclerosis (ALS) market is experiencing steady growth, with a projected CAGR of approximately 7-8% during the forecast period. This growth is driven by increasing disease awareness, a rising number of ALS cases globally, growing research investments aimed at discovering effective treatments, advancements in biotechnology, expanding clinical trials, supportive government initiatives, and the development of innovative therap…

Ractigen's RAG-17 ALS Data Shines at AAN 2025: Earns Top Award, Phase I Advances

Final results from an Investigator-Initiated Trial presented for the first time, demonstrating favorable safety, significant target engagement, and encouraging clinical & biomarker signalsAbstract honored with prestigious "Abstracts of Distinction" award by AAN Science Committee, recognizing top quality and interestPhase I…