AAV9 based ChAT gene therapy (congenital myasthenic syndrome)

Pyridostigmine(Mestinon, Regonol)

C₉H₁₃N₂O₂ · Rx

congenital hyperinsulinism therapeutic

congenital liver disease therapy

Leigh syndrome therapeutic(Leigh syndrome)

ORAI-XXXX

Haemorrhagic Fever with Renal Syndrome Bivalent Vaccine (Vero Cell),Inactivated

Rett Syndrome (ACADIA)

Rett syndrome therapy

Sjogren's syndrome (Precisionlife)