Nitisinone(Nityr, Orfadin)

C₁₄H₁₀F₃NO₅ · Rx

RNA actuator therapeutic for hereditary angioedema(CAMP4 Therapeutics)

RNA actuator therapeutic (hereditary angioedema)

C1 esterase inhibitor (hereditary angioedema)

Hereditary muscle disease(Belief BioMed)

Hereditary neurological intractable diseases(Luxna)

agonist antibody (hereditary hemorrhagic telangiectasia)

FAH gene therapy (lentiviral vector, tyrosinemia type I )

Hereditary angioedema gene therapy (Spark Therapeutics)

AP4M1 gene therapy (AAV9, hereditary spastic paraplegia 50)