The ALS Puzzle Gets Two New Pieces: SYF2 and PIKFYVE

ALS is the 5,000-piece jigsaw puzzle of the therapeutic world. As the regulatory edges come together, new research pieces are also connecting.

In 2022, the association between gene-encoding protein UNC13A and the TDP-43 protein – long implicated in ALS pathology – was discovered by University College London and the NIH. More recently, attention has fallen to two potential therapeutic targets: PIKFYVE and SYF2.