**Rare neurodeg RNA/ASO/ERT approvals + new disorders + AI dx + LSD small-mols [climaxing]** [climaxing]
Key Questions
What is Zycubo approved for?
FDA approved Zycubo for Menkes disease, alongside Avlayah, marking RNA/ASO/ERT approvals for rare neurodeg disorders.
What is zilganersen for AxD?
Zilganersen is an ASO therapy approved for Alexander disease (AxD), part of surging approvals for rare neurodegeneration.
What is venglustat's status for GD3?
Venglustat for Gaucher disease type 3 (GD3) received BTD and is advancing past Ph3 compared to ERT.
What are recent approvals like Daybue and VYKAT?
Daybue for Rett syndrome; VYKAT for Prader-Willi syndrome (PWS); Spinraza/salanersen and relutrigine for DEE.
What is Boca GENA's AI for rare neurodeg?
Boca GENA uses AI for diagnosing rare neurodegenerative diseases, inspired by yearslong diagnostic waits.
FDA Zycubo Menkes Avlayah; venglustat GD3 BTD Ph3>ERT; TK2d; zilganersen AxD; Daybue Rett; Spinraza/salanersen; relutrigine DEE; VYKAT PWS; Polaryx PLX-200 Ph2; Monopar ALXN1840 Wilson Ph3 NDA mid'26; Boca GENA AI dx rare neurodeg.