Genes associated with “progressive neurologic deterioration

89 genes foundHPO: Progressive neurologic deteriorationOpen Targets: Progressive neurologic deterioration3 PanelApp panels
How are genes scored? (0–100 composite)
-5–15
ClinGen
0–15
HPO Freq
0–15
Open Targets
0–12
Phen2Gene
0–10
ClinVar
0–8
Constraint
0–8
Dosage
0–8
OMIM CS
0–8
PanelApp
0–5
OMIM
0–5
G2P
0–4
Breadth
Tiers:Strong Candidates (≥20)Consider (≥8)Possible (≥3)
Evidence dots:
HPOClinVarPhen2GeneOpen TargetsPanelApp
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Strong Candidates

2 genes
31
score
ClinGen: DefinitiveP2G #40GTR ↑
Frequency
-
P/LP Variants
33
OT Score
-
23
score
ClinGen: DefinitiveP2G #1GTR ↑

myoclonic epilepsy of Lafora 2

Frequency
82%
n=22
P/LP Variants
-
OT Score
-

Consider

19 genes
17ADSL
Def

adenylosuccinate lyase

15EPM2A
Def#2

myoclonic epilepsy of Lafora 1

14SYNJ1
Def#20

developmental and epileptic encephalopathy, 53

14MECP2
Def

methyl-CpG binding protein 2

13FOLR1
Def

NEUROLOGIC, ENDOCRINE, AND PANCREATIC DISEASE, MULTISYSTEM, INFANTILE-ONSET 1; IMNEPD1

LEUKOENCEPHALOPATHY, PROGRESSIVE, INFANTILE-ONSET, WITH OR WITHOUT DEAFNESS; LEPID

LEUKOENCEPHALOPATHY, DEVELOPMENTAL DELAY, AND EPISODIC NEUROLOGIC REGRESSION SYNDROME; LEUDEN

13MTHFR
Def
13PDHA1
Def
13SLC17A5
Def
13KCNJ11
Def

Diabetes mellitus, transient neonatal 3

skeletal overgrowth-craniofacial dysmorphism-hyperelastic skin-white matter lesions syndrome

11SUMF1
Def#3

mucosulfatidosis

10IDUA
Def#4

Hurler syndrome

9CERS1
Mod

progressive myoclonic epilepsy type 8

9CNTNAP2
Def#6

cortical dysplasia-focal epilepsy syndrome

9ATP7A
Def
9HSD17B10
Def#19

HSD10 mitochondrial disease

Possible

53 genes — click to expand
8GCH1
Def

GTP cyclohydrolase I deficiency with hyperphenylalaninemia

8PSAP
Def#9

Krabbe disease due to saposin A deficiency

8LMNB1
Def

leukodystrophy, demyelinating, adult-onset, autosomal dominant, typical

7GLB1
Def#7

GM1 gangliosidosis type 2

Hemolytic anemia, CD59-mediated, with or without immune-mediated polyneuropathy

7CLN3
Def
7EHMT1
Def
7MTO1
Def
7MYH9
Def
7PDHX
Def
7RAG2
Def
7HEXB
Def#8

Sandhoff disease

6EIF2B5
Def

leukoencephalopathy with vanishing white matter 5

6RAB27A
Def#10

Griscelli syndrome type 2

6NAGLU
Def#11

mucopolysaccharidosis type 3B

6MTFMT
Def

combined oxidative phosphorylation defect type 15

6TINF2
Def#17

Revesz syndrome

6PTS
Def

BH4-deficient hyperphenylalaninemia A

6QDPR
Def

dihydropteridine reductase deficiency

6CTNS
Def

nephropathic cystinosis

6MCOLN1
Def

mucolipidosis type IV

mitochondrial complex I deficiency, nuclear type 31

6GRN
Def

GRN-related frontotemporal lobar degeneration with Tdp43 inclusions

megalencephalic leukoencephalopathy with subcortical cysts 2A

Leukodystrophy, demyelinating, adult-onset, autosomal dominant, typical

AICARDI-GOUTIERES SYNDROME 9; AGS9

5CLPB
Lim

GAUCHER DISEASE, TYPE II; GD2

HYPERPHENYLALANINEMIA, BH4-DEFICIENT, A; HPABH4A

HYPERPHENYLALANINEMIA, BH4-DEFICIENT, B; HPABH4B

HYPERPHENYLALANINEMIA, BH4-DEFICIENT, C; HPABH4C

MUCOLIPIDOSIS IV; ML4

5NOTCH3
Lim

notch receptor 3

5NDUFA6
Mod#18

mitochondrial complex I deficiency, nuclear type 33

combined oxidative phosphorylation deficiency 37

4ERCC6
Def

cerebrooculofacioskeletal syndrome 1

microcephalic osteodysplastic primordial dwarfism type I

3CYC1
Mod

mitochondrial complex III deficiency nuclear type 6

3MT-CO2
Def

mitochondrially encoded cytochrome c oxidase II

3TBXAS1
Def

thromboxane A synthase 1

3GBA1
Def

Gaucher disease perinatal lethal

Data aggregated from HPO/Monarch, ClinVar, OMIM (phenotypes + clinical synopsis), Open Targets, PanelApp, Phen2Gene, and Gene2Phenotype. Click a gene to view full details.