Klotho Neurosciences is advancing KLTO‑202, its gene therapy candidate for amyotrophic lateral sclerosis (ALS), to the manufacturing and process development phase — with an eye toward trials in humans beginning next year. The manufacturing phase is expected to last about eight months, and will be followed by 4-6…
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Sineugene Therapeutics‘ SNUG01, a gene therapy for people with amyotrophic lateral sclerosis (ALS). The designation is given to potential treatments for rare diseases, or those affecting fewer than 200,000 people in the U.S. It provides…
Clene has incorporated suggestions from the U.S. Food and Drug Administration (FDA) on its analysis plans for certain biomarker data that’s expected to support an accelerated approval application for CNM-Au8 to treat amyotrophic lateral sclerosis (ALS), the company has announced. Specifically, the FDA had suggested revisions to…
Mindy Uhrlaub, an author and activist for amyotrophic lateral sclerosis (ALS), has spent a good part of her life caring for loved ones. Soon after she became a mom in 2003, her husband learned he had cancer. “Right as my older son was taking his first wobbly steps, my…
The Longitude Prize on ALS is offering a total of £7.5 million (more than $10 million) to encourage scientists to use artificial intelligence (AI) to speed up the search for new treatments for amyotrophic lateral sclerosis (ALS), The entry window closes in early December. Over five years,…
Living close to water bodies with blue-green algae blooms, or cyanobacteria, is linked to significantly reduced survival among people with amyotrophic lateral sclerosis (ALS), according to a new study. The association was significantly stronger in people who used private well water or participated in water-related activities such as swimming…
Three years after its approval in the U.S., Radicava ORS (edaravone) has been prescribed by more than 1,300 healthcare providers to more than 13,500 people living with amyotrophic lateral sclerosis (ALS) in the country, according to the company that markets it. First approved as an into-the-vein infusion called…
Clinicians view early access to palliative care as beneficial for people with amyotrophic lateral sclerosis (ALS), yet its integration into routine care remains inconsistent, a U.S. study found. About two-thirds of ALS clinicians said they had a palliative care specialist on their team, and just over half of ALS…
An experimental oral treatment from Zydus Lifesciences was well tolerated at all tested doses and showed signs of slowing disease progression in people with amyotrophic lateral sclerosis (ALS). That’s according to findings from a proof-of-concept Phase 2a trial (NCT05981040) in India that tested Zydus’ usnoflast in 24…
Nine out of 10 people with amyotrophic lateral sclerosis (ALS) who received the experimental cell therapy NurOwn (debamestrocel) in an expanded access program (EAP) following a placebo-controlled Phase 3 trial survived for at least five years after the onset of their symptoms. That’s according to data announced by…
