Israel’s Neuromagen (see here previously) has received US FDA Orphan designation for its AGS-499 candidate treatment of ALS (Amyotrophic Lateral Sclerosis), also known as Lou Gehrig’s disease. AGS-499 has shown outstanding promise in lab tests, including using human cells.

https://www.biospace.com/article/neuromagen-pharma-ltd-receives-fda-orphan-drug-designation-for-its-ags-499-drug-candidate-to-slow-the-progression-of-als-amyotrophic-lateral-sclerosis-/