List view / Grid view
With the emergence of innovative therapeutic technologies and progress in the genetic understanding of diseases, Biogen’s Toby Ferguson explains why it is a promising time for the development of therapies for neuromuscular conditions such as amyotrophic lateral sclerosis (ALS).
The EMA has accepted the marketing authorisation application for tofersen, which could be the first treatment for a rare genetic cause of ALS.
Research for rare diseases including ALS will be supported over the next four years by $38 million in FDA funding.
Sanofi’s Japan-based manufacturing site near Tokyo will be sold to a European contract development and manufacturing organisation.
The Critical Path for Rare Neurodegenerative Diseases (CP-RND) partnership was launched to develop breakthrough treatments for neurodegenerative diseases like amyotrophic lateral sclerosis (ALS).
Amyotrophic lateral sclerosis (ALS) patients treated with AstroRx® had a 45 percent reduction in their disease progression rate.
The data suggests disease deterioration was halved in the six Amyotrophic Lateral Sclerosis (ALS) patients treated with IPL344 over eight months.
Tofersen reduced expression of the SOD1 gene known to cause amyotrophic lateral sclerosis (ALS) and slowed clinical decline in the Phase I/II trial.
The FDA has granted early-action approval to Exservan for the treatment of ALS, which can be administered safely and easily without water.
The FDA has handed a CRL to Biohaven for its ALS treatment awaiting approval due to concerns regarding the medication’s API.
