PAH

Phenylalanine-4-hydroxylase · P00439 · PAH on Sugi Atlas →

170 patent compounds predicted against PAH, 132 high-confidence (nearest known ligand ≥ 0.5)

Top predicted compounds (top 20 by supporting neighbours, then confidence)

Compound	Confidence	Supporting neighbours
Sapropterin (SCHEMBL260185)	0.81	1/20
Sapropterin (SCHEMBL4217501)	0.81	1/20
Sapropterin (SCHEMBL7942378)	0.81	1/20
Sapropterin (SCHEMBL14072498)	0.81	1/20
Sapropterin (SCHEMBL14401407)	0.81	1/20
Sapropterin (SCHEMBL21068033)	0.81	1/20
Sapropterin (SCHEMBL29059706)	0.81	1/20
Sapropterin (SCHEMBL29351671)	0.81	1/20
Sapropterin (SCHEMBL29538547)	0.81	1/20
Sapropterin (SCHEMBL1070424)	0.79	1/20
Sapropterin (SCHEMBL29406211)	0.79	1/20
Sapropterin (SCHEMBL31106620)	0.79	1/20
Sapropterin (SCHEMBL28059757)	0.67	1/20
Sapropterin (SCHEMBL37480)	0.62	1/20
Sapropterin (SCHEMBL258544)	0.62	1/20
Sapropterin (SCHEMBL1534380)	0.62	1/20
Sapropterin (SCHEMBL20769839)	0.62	1/20
Sapropterin (SCHEMBL25915067)	0.62	1/20
Sapropterin (SCHEMBL25915163)	0.62	1/20
Sapropterin (SCHEMBL28826011)	0.62	1/20

Clinical trials — most relevant to PAH by text similarity (a relevance ranking, not an exhaustive list)

Trial	Phase	Status
Effects of Kuvan on Melatonin Secretion	PHASE4	COMPLETED
The Effectiveness of Kuvan in Amish PKU Patients	PHASE4	COMPLETED
BH4 Responsiveness in PAH Deficiency PKU Patients	PHASE4	UNKNOWN
Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients	PHASE3	COMPLETED
The Efficacy and Safety of Vardenafil in the Treatment of Pulmonary Arterial Hypertension	PHASE3	COMPLETED
A Study of Selexipag as Add-On Treatment to Standard of Care in Children With Pulmonary Arterial Hypertension	PHASE3	ACTIVE_NOT_RECRUITING
Iron Supplements Effect on b-Phe Levels in PKU Patients During 3 Years Study	PHASE2	COMPLETED
Study of BH4, a New and Simple Treatment of Mild PKU	PHASE2	COMPLETED
A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia	PHASE1/PHASE2	COMPLETED
Safety and Efficacy Study of NGGT002 in cPKU Adult Subjects	PHASE1/PHASE2	RECRUITING
Bardoxolone Methyl Evaluation in Patients With Pulmonary Hypertension (PH) - LARIAT	PHASE2	COMPLETED
A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria	PHASE1	RECRUITING
Early Dietary Treated Patients With Phenylketonuria Can Achieve Normal Growth and Mental Development.	nan	UNKNOWN
Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)	nan	RECRUITING
Comparison of Atherogenic Risk Factors and Efficacy of Nutritional Treatment Among Adult Phenylketonuria Patients	nan	NOT_YET_RECRUITING
Nutritional and Neurotransmitter Changes in PKU Subjects on BH4	nan	COMPLETED
Biological Variation of Phenylalanine in Patients With Hyperphenylalaninemia	nan	COMPLETED
Low Phenylalanine Diet for Mothers With Phenylketonuria (PKU)	nan	COMPLETED
SNAP: Study Nutrients in Adult PKU	nan	COMPLETED
Observational Study of Endothelial Dysfunction in Phenylketonuria	nan	COMPLETED
Fluorodeoxyglucose Positron Emission Tomography (FDG PET) Findings in Patients With Phenylketonuria Before and After KUVAN Therapy	nan	COMPLETED
PANDA: PKU Amino Acid Evaluation	nan	COMPLETED
A Single Patient Study of Sapropterin for Multisystem Smooth Muscle Dysfunction Syndrome	nan	NOT_YET_RECRUITING
The Brain and Neuropsychological Functioning in Adults With Sapropterin Dihydrochloride Treated Phenylketonuria	nan	COMPLETED
Study of Low-grade Systemic Inflammation in Adult Patients With Phenylketonuria	nan	COMPLETED
Genes Associated With Development of Pulmonary Arterial Hypertension in Patients With Congenital Shunt Lesions	nan	RECRUITING
The Effect of Nursing Empowerment Program	nan	COMPLETED
Protein Requirements in Adults With Phenylketonuria (PKU)	nan	COMPLETED
Biomarker for the Early Diagnosis and Monitoring in Tyrosinemia Type 1 (BioTyrosin)	nan	WITHDRAWN
BMPR2 Mutations and Iron Metabolism in Pulmonary Arterial Hypertension	nan	COMPLETED

Related proteins — ESM-2 sequence neighbours (a PAH binder may also engage these)

Protein	Name	Similarity
TPH1	Tryptophan 5-hydroxylase 1	1.000	landscape →
P16331	—	1.000	P16331 →
P17290	—	1.000	P17290 →
P09810	—	1.000	P09810 →
P04176	—	1.000	P04176 →
P17276	—	1.000	P17276 →
P70080	—	1.000	P70080 →
Q2KIH7	—	1.000	Q2KIH7 →
Q2HZ26	—	1.000	Q2HZ26 →
P11982	—	1.000	P11982 →
Q8CGU9	—	0.999	Q8CGU9 →
Q54XS1	—	0.999	Q54XS1 →
E5KBU3	—	0.999	E5KBU3 →
TPH2	Tryptophan 5-hydroxylase 2	0.999	landscape →
Q92142	—	0.998	Q92142 →
P90925	—	0.997	P90925 →
P17532	—	0.997	P17532 →
P90986	—	0.997	P90986 →
A8X3V8	—	0.997	A8X3V8 →
A0A060X6Z0	—	0.996	A0A060X6Z0 →
Q0EAB8	—	0.996	Q0EAB8 →
Q8CGV2	—	0.995	Q8CGV2 →
P17289	—	0.994	P17289 →
P24529	—	0.994	P24529 →
Q9STV0	—	0.994	Q9STV0 →
Q76IQ3	—	0.993	Q76IQ3 →
P04177	—	0.993	P04177 →
Q9USN5	—	0.992	Q9USN5 →
P00365	—	0.991	P00365 →
Q8L831	—	0.991	Q8L831 →