amyotrophic lateral sclerosis

Apr 25, 2025

The 77th Annual Meeting of the American Academy of Neurology (AAN 2025), held from April 5–9 in San Diego and online, served as a pivotal forum for advancing research in neurology, with particular relevance to neurodegenerative diseases. As one of the largest gatherings of neurologists globally, the conference prov...

Nov 26, 2024

Ractigen Therapeutics Receives FDA Orphan Drug Designation for RAG-21 to Treat ALS Ractigen Therapeutics has announced that the FDA has granted Orphan Drug Designation (ODD) to RAG-21, its novel siRNA therapy for treating amyotrophic lateral sclerosis (ALS). RAG-21 specifically targets FUS-ALS, one of the most a...

Sep 24, 2024

The amyotrophic lateral sclerosis treatment landscape is stringent and includes multidisciplinary care, such as physical therapy, speech therapy, dietary counseling, heat or whirlpool therapy, and others. The approved drugs for amyotrophic lateral sclerosis treatment include RILUZOLE, RADICAVA, TIGLUTIK, EXSERVAN, ...

Sep 24, 2024

DelveInsight forecasts that with the launch of 15+ emerging therapies alongside existing treatments, the ALS treatment market is anticipated to surpass USD 4 billion across the 7MM by 2034. Currently, there is no cure for ALS and no effective treatment to halt or reverse the progression of the disease. As per ou...

Sep 24, 2024

Amyotrophic Lateral Sclerosis (ALS) or Lou Gehrig’s disease is a rare, progressive, and fatal neuromuscular disease characterized by degeneration of upper and lower motor neurons. While it is associated with a mortality rate of 50% within 30 months of Amyotrophic Lateral Sclerosis symptoms onset, there is still no ...

Sep 24, 2024

Amyotrophic Lateral Sclerosis (ALS) is a devastating neurodegenerative disease characterized by the progressive degeneration of motor neurons, leading to muscle weakness, paralysis, and ultimately, death. Over the past decade, a growing understanding of the disease's underlying mechanisms has led to significant adv...

Feb 27, 2024

Survodutide Phase II trial Shows Groundbreaking Results in Liver Disease due to MASH, with Significant Improvements in Fibrosis Boehringer Ingelheim has reported that in a Phase II trial, a significant proportion of adults treated with survodutide (BI 456906), up to 83.0%, showed a notable enhancement in metabol...

May 02, 2023

FDA Grants Priority Review to Luspatercept for First-line Treatment of Anemia in Lower-risk MDS The FDA has granted priority review to a supplemental biologics license application (sBLA) seeking to expand the current indication of luspatercept-aamt (Reblozyl) to include treatment of anemia in patients with very ...

Apr 04, 2023

HUTCHMED Completes Rolling Submission of NDA to FDA for Fruquintinib HUTCHMED (China) Limited announced the completion of the rolling submission of a New Drug Application ("NDA") to the United States Food and Drug Administration ("FDA") for fruquintinib, its highly selective and potent oral inhibitor of VEGFR-1,...

Feb 28, 2023

Sage Therapeutics Receives EU Orphan Drug Designation for Huntington's Disease Treatment Candidate Sage Therapeutics declared the European Medicines Agency granted Orphan Drug Designation to SAGE-718 for the treatment of Huntington's disease. SAGE-718 is developing as a potential oral therapy for cognitive disor...