Hereditary pulmonary alveolar proteinosis

Summary

Hereditary pulmonary alveolar proteinosis (MONDO:0012580) is a disease (an umbrella term covering 8 Mondo subtypes) with 6 cohort genes and 2 clinical trials. The dominant Reactome pathway is Defective CSF2RB causes SMDP5 (4 cohort genes). Top therapeutic interventions include sargramostim.

At a glance

• Prevalence: <1 / 1 000 000 (Worldwide) [Orphanet-validated]
• Umbrella term: 8 Mondo subtypes
• Cohort genes: 6
• ClinVar variants: 781
• Phenotypes (HPO): 15
• Clinical trials: 2

Clinical features

Epidemiology

Prevalence records

1 prevalence record(s), Orphanet:

Type	Class	Value	Geography	Validation
Point prevalence	<1 / 1 000 000		Worldwide	Validated

Signs & symptoms

Clinical features (HPO)

15 HPO clinical features (Orphanet curated; top 15 by frequency):

HPO ID	Term	Frequency
HP:0010876	Abnormal circulating protein level	Very frequent (80-99%)
HP:0001531	Failure to thrive in infancy	Frequent (30-79%)
HP:0002091	Restrictive ventilatory defect	Frequent (30-79%)
HP:0002098	Respiratory distress	Frequent (30-79%)
HP:0004887	Respiratory failure requiring assisted ventilation	Frequent (30-79%)
HP:0012418	Hypoxemia	Frequent (30-79%)
HP:0025391	Crazy paving pattern on pulmonary HRCT	Frequent (30-79%)
HP:0001649	Tachycardia	Occasional (5-29%)
HP:0002789	Tachypnea	Occasional (5-29%)
HP:0003651	Foam cells	Occasional (5-29%)
HP:0011949	Acute infectious pneumonia	Occasional (5-29%)
HP:0012735	Cough	Occasional (5-29%)
HP:0030057	Autoimmune antibody positivity	Occasional (5-29%)
HP:0030830	Crackles	Occasional (5-29%)
HP:0031029	Elevated carcinoembryonic antigen level	Occasional (5-29%)

Identifiers

Disease identifiers

Field	Value
Canonical name	hereditary pulmonary alveolar proteinosis
Mondo ID	MONDO:0012580
MeSH	C535832
OMIM	265120
Orphanet	264675
SNOMED CT	707442002
UMLS	C3711368
MedGen	777976
GARD	0004582
Is cancer (heuristic)	no

Also known as: congenital PAP · congenital pulmonary alveolar proteinosis · hereditary pulmonary alveolar proteinosis · inborn error of pulmonary surfactant metabolism · inborn error of surfactant metabolism · pulmonary alveolar proteinosis, congenital

Data availability: 781 ClinVar variants · 3 GenCC gene-disease records.

Disease family

An umbrella term covering 8 Mondo subtypes.

Classification path: disease › human disease › disease by body system or component › respiratory system disorder › lower respiratory tract disorder › lung disorder › pulmonary alveolar proteinosis › hereditary pulmonary alveolar proteinosis

Related subtypes (2): autoimmune pulmonary alveolar proteinosis, secondary pulmonary alveolar proteinosis

Subtypes (8): surfactant metabolism dysfunction, pulmonary, 1, surfactant metabolism dysfunction, pulmonary, 4, interstitial lung disease due to ABCA3 deficiency, surfactant metabolism dysfunction, pulmonary, 5, severe early-onset pulmonary alveolar proteinosis due to MARS deficiency, chronic respiratory distress with surfactant metabolism deficiency, SFTPC-related interstitial lung disease, surfactant metabolism dysfunction, pulmonary, 2

Genetics & variants

GWAS landscape

No GWAS associations recorded — common-variant (GWAS) studies don’t cover this disease (typical for Mendelian / rare diseases). See the curated gene cohort and Mendelian overlap below.

Variant details and genetic-evidence tiers

ClinVar germline variants

600 retrieved; paginated sample, class counts are floors:

295 uncertain significance, 142 likely benign, 75 conflicting classifications of pathogenicity, 26 benign/likely benign, 26 benign, 18 pathogenic, 9 pathogenic/likely pathogenic, 9 likely pathogenic

ClinVar	Variant (HGVS)	Gene	Classification	Review
1068619	NM_001089.3(ABCA3):c.1729_1730del (p.Ser577fs)	ABCA3	Pathogenic	criteria provided, multiple submitters, no conflicts
1211382	NM_001089.3(ABCA3):c.3863-98C>T	ABCA3	Pathogenic	criteria provided, multiple submitters, no conflicts
1317554	NM_001089.3(ABCA3):c.3997_3998del (p.Arg1333fs)	ABCA3	Pathogenic	criteria provided, multiple submitters, no conflicts
1331730	NM_001089.3(ABCA3):c.4141_4142del (p.Leu1381fs)	ABCA3	Pathogenic	criteria provided, multiple submitters, no conflicts
1730473	NM_001089.3(ABCA3):c.3348_3360del (p.Ile1117fs)	ABCA3	Pathogenic	criteria provided, single submitter
1735105	NM_001089.3(ABCA3):c.3805G>T (p.Glu1269Ter)	ABCA3	Pathogenic	criteria provided, single submitter
1736036	NM_001089.3(ABCA3):c.3902dup (p.Val1303fs)	ABCA3	Pathogenic	criteria provided, multiple submitters, no conflicts
1738591	NM_001089.3(ABCA3):c.4195G>A (p.Val1399Met)	ABCA3	Pathogenic	criteria provided, multiple submitters, no conflicts
1740383	NM_001089.3(ABCA3):c.440C>T (p.Pro147Leu)	ABCA3	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
1758921	NM_001089.3(ABCA3):c.743C>T (p.Pro248Leu)	ABCA3	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
1767530	NM_001089.3(ABCA3):c.127C>T (p.Arg43Cys)	ABCA3	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
1769442	NM_001089.3(ABCA3):c.1302G>A (p.Trp434Ter)	ABCA3	Pathogenic	criteria provided, single submitter
1772770	NM_001089.3(ABCA3):c.1444C>T (p.Gln482Ter)	ABCA3	Pathogenic	criteria provided, single submitter
1773031	NM_001089.3(ABCA3):c.1455C>A (p.Tyr485Ter)	ABCA3	Pathogenic	criteria provided, single submitter
1773032	NM_001089.3(ABCA3):c.1455C>G (p.Tyr485Ter)	ABCA3	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
1799308	NM_001089.3(ABCA3):c.3057dup (p.Asn1020Ter)	ABCA3	Pathogenic	criteria provided, single submitter
203381	NM_001089.3(ABCA3):c.875A>T (p.Glu292Val)	ABCA3	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
228321	NM_001089.3(ABCA3):c.128G>A (p.Arg43His)	ABCA3	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
2982329	NM_001089.3(ABCA3):c.737C>T (p.Pro246Leu)	ABCA3	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
13201	NM_000542.5(SFTPB):c.361delinsGAA (p.Pro121fs)	SFTPB	Pathogenic	criteria provided, multiple submitters, no conflicts
1752055	NM_000542.5(SFTPB):c.581del (p.Gln194fs)	SFTPB	Pathogenic	criteria provided, single submitter
1761177	NM_000542.5(SFTPB):c.754C>T (p.Arg252Cys)	SFTPB	Pathogenic	criteria provided, single submitter
1766142	NM_000542.5(SFTPB):c.883C>T (p.Arg295Ter)	SFTPB	Pathogenic	criteria provided, single submitter
13208	NM_001317778.2(SFTPC):c.218T>C (p.Ile73Thr)	SFTPC	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
1729939	NM_001317778.2(SFTPC):c.329T>C (p.Leu110Pro)	SFTPC	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
1738082	NM_001317778.2(SFTPC):c.413_430delinsAGGTGATC (p.Thr138fs)	SFTPC	Pathogenic	criteria provided, single submitter
1744578	NM_001317778.2(SFTPC):c.480dup (p.Arg161fs)	SFTPC	Pathogenic	criteria provided, single submitter
1740119	NM_001089.3(ABCA3):c.4376G>A (p.Gly1459Asp)	ABCA3	Likely pathogenic	criteria provided, multiple submitters, no conflicts
1741928	NM_001089.3(ABCA3):c.4618G>A (p.Glu1540Lys)	ABCA3	Likely pathogenic	criteria provided, multiple submitters, no conflicts
1742161	NM_001089.3(ABCA3):c.4648C>T (p.Arg1550Trp)	ABCA3	Likely pathogenic	criteria provided, multiple submitters, no conflicts

Genes & proteins

Mendelian disease overlap and somatic drivers

GenCC: 7 · Orphanet: 12 · OMIM-shared: 0 · Dual-evidence (GWAS+Mendelian): 0

GenCC gene–disease validity (cohort genes)

the Disease column is the GenCC-asserted condition — a cohort gene’s strongest validity may be for a related predisposition syndrome.

Gene	Classification	Inheritance	Disease	Records
CSF2RA	Definitive	Autosomal recessive	surfactant metabolism dysfunction, pulmonary, 4	3
CSF2RB	Moderate	Autosomal recessive	surfactant metabolism dysfunction, pulmonary, 5	3
ACADL	Limited	Autosomal recessive	hereditary pulmonary alveolar proteinosis

Orphanet rare-disease linkage (cohort genes)

Gene	Orphanet ID	Rare disease
CSF2RA	Orphanet:264675	Hereditary pulmonary alveolar proteinosis
CSF2RB	Orphanet:264675	Hereditary pulmonary alveolar proteinosis
SFTPB	Orphanet:217563	Neonatal acute respiratory distress syndrome
SFTPB	Orphanet:685082	Pediatric acute respiratory distress syndrome
SFTPC	Orphanet:2032	Idiopathic pulmonary fibrosis
SFTPC	Orphanet:217566	Chronic respiratory distress with surfactant metabolism deficiency
SFTPC	Orphanet:440392	Interstitial lung disease due to SP-C deficiency
SFTPC	Orphanet:685082	Pediatric acute respiratory distress syndrome
ABCA3	Orphanet:2032	Idiopathic pulmonary fibrosis
ABCA3	Orphanet:217563	Neonatal acute respiratory distress syndrome
ABCA3	Orphanet:440402	Interstitial lung disease due to ABCA3 deficiency
ABCA3	Orphanet:685082	Pediatric acute respiratory distress syndrome

Cohort genes → proteins

6 cohort genes, 6 distinct canonical proteins.

Evidence partition

Subset	Genes
multi_evidence	6

Cohort genes (full)

Symbol	HGNC	Ensembl	UniProt	Name	Evidence
CSF2RA	HGNC:2435	ENSG00000198223	P15509	Granulocyte-macrophage colony-stimulating factor receptor subunit alpha	gencc
CSF2RB	HGNC:2436	ENSG00000100368	P32927	Cytokine receptor common subunit beta	gencc
ACADL	HGNC:88	ENSG00000115361	P28330	Long-chain specific acyl-CoA dehydrogenase, mitochondrial	gencc
SFTPB	HGNC:10801	ENSG00000168878	P07988	Pulmonary surfactant-associated protein B	clinvar
SFTPC	HGNC:10802	ENSG00000168484	P11686	Surfactant protein C	clinvar
ABCA3	HGNC:33	ENSG00000167972	Q99758	Phospholipid-transporting ATPase ABCA3	clinvar

Cohort function summary

Lead sentence per gene, UniProt-curated.

Symbol	Protein name	Function (lead sentence)
CSF2RA	Granulocyte-macrophage colony-stimulating factor receptor subunit alpha	Low affinity receptor for granulocyte-macrophage colony-stimulating factor.
CSF2RB	Cytokine receptor common subunit beta	Cell surface receptor that plays a role in immune response and controls the production and differentiation of hematopoietic progenitor cells into lineage-restricted cells.
ACADL	Long-chain specific acyl-CoA dehydrogenase, mitochondrial	Long-chain specific acyl-CoA dehydrogenase is one of the acyl-CoA dehydrogenases that catalyze the first step of mitochondrial fatty acid beta-oxidation (FAO), breaking down fatty acids into acetyl-CoA and allowing the production of energy…
SFTPB	Pulmonary surfactant-associated protein B	Pulmonary surfactant-associated proteins promote alveolar stability by lowering the surface tension at the air-liquid interface in the peripheral air spaces.
SFTPC	Surfactant protein C	Pulmonary surfactant associated proteins promote alveolar stability by lowering the surface tension at the air-liquid interface in the peripheral air spaces.
ABCA3	Phospholipid-transporting ATPase ABCA3	Catalyzes the ATP-dependent transport of phospholipids such as phosphatidylcholine and phosphoglycerol from the cytoplasm into the lumen side of lamellar bodies, in turn participates in the lamellar bodies biogenesis and homeostasis of pul…

Protein-family classification

Druggable: 3 · Difficult: 0 · Unknown: 3 · Druggable fraction: 0.5

Family distribution

Cohort families vs a genome-wide background (hypergeometric, BH-FDR; fold = observed/expected). Counts kept; sorted by enrichment, so the catch-all Other/Unknown bucket no longer leads.

Family	Genes	Fold	FDR
Antibody/Immunoglobulin	2	9.7×	0.048
Transporter	1	13.0×	0.112
Other/Unknown	3	0.9×	0.758

Per-gene assignment

Symbol	Family	Druggable?	EC	InterPro (top 3)
CSF2RA	Antibody/Immunoglobulin	yes		Short_hematopoietin_rcpt_2_CS, FN3_dom, Ig-like_fold
CSF2RB	Antibody/Immunoglobulin	yes		Hempt_rcpt_S_F1_CS, FN3_dom, IL3_rcpt_beta
ACADL	Other/Unknown	no		Acyl-CoA_DH_CS, AcylCoA_DH/ox_M, AcylCo_DH/oxidase_C
SFTPB	Other/Unknown	no		SAP_A, SapB_1, SapB_2
SFTPC	Other/Unknown	no		SP-C, BRICHOS_dom, Surfactant_protein_propep
ABCA3	Transporter	yes		ABC_transporter-like_ATP-bd, AAA+_ATPase, ABC2_TM

Expression context

Cohort genes with no expression data: 0.

6 cohort genes are a single-cell marker in ≥1 SCXA experiment.

Breadth distribution (Bgee present_calls)

Bucket	Genes
narrow (1-5 tissues)	0
moderate (6-20)	0
broad (>20)	6
unknown	0

Top tissues across cohort

Tissue	Cohort genes
lower lobe of lung	3
upper lobe of lung	2
leukocyte	1
monocyte	1
mononuclear cell	1
blood	1
decidua	1
periodontal ligament	1
body of pancreas	1
popliteal artery	1
tibial artery	1
visceral pleura	1
adult organism	1
right lung	1
upper lobe of left lung	1

Per-gene tissue summary (top 30)

Symbol	Bgee breadth	FANTOM5 breadth	SCXA	Top tissues
CSF2RA	193		marker	monocyte, mononuclear cell, leukocyte
CSF2RB	230	broad	marker	blood, periodontal ligament, decidua
ACADL	208	broad	marker	body of pancreas, popliteal artery, tibial artery
SFTPB	231	tissue_specific	marker	lower lobe of lung, visceral pleura, upper lobe of lung
SFTPC	208	tissue_specific	marker	lower lobe of lung, right lung, adult organism
ABCA3	222	ubiquitous	marker	lower lobe of lung, upper lobe of lung, upper lobe of left lung

Protein interactions among cohort

Intra-cohort edges: 7.

Hub genes (top 10 by interactor count)

Symbol	Interactor count
ACADL	2,251
CSF2RB	1,948
SFTPC	1,613
ABCA3	1,436
CSF2RA	1,335
SFTPB	899

Intra-cohort edges

A	B	Sources
ABCA3	CSF2RA	string_interaction
ABCA3	CSF2RB	string_interaction
ABCA3	SFTPB	string_interaction
ABCA3	SFTPC	string_interaction
CSF2RA	CSF2RB	string_interaction
CSF2RA	SFTPB	string_interaction
SFTPB	SFTPC	string_interaction

Structural data

PDB: 6 · AlphaFold-only: 0 · No structure: 0

Cohort genes with PDB structures (top 30)

Symbol	UniProt	PDB entries
CSF2RB	P32927	10
SFTPB	P07988	9
ACADL	P28330	3
SFTPC	P11686	3
CSF2RA	P15509	2
ABCA3	Q99758	2

Function

Pathway analysis

Distinct Reactome pathways touched by cohort: 27. Enrichment computed across 6 evidence-associated genes (6 with Reactome annotation).

Pathways by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 6 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

Pathway	Cohort genes	Fold	FDR	Sample cohort genes
Defective CSF2RB causes SMDP5	4	1087.6×	7e-12	SFTPB, SFTPC, CSF2RA, CSF2RB
Defective CSF2RA causes SMDP4	4	1087.6×	7e-12	SFTPB, SFTPC, CSF2RA, CSF2RB
Surfactant metabolism	5	307.0×	7e-12	SFTPB, SFTPC, CSF2RA, CSF2RB, ABCA3
Interleukin receptor SHC signaling	2	135.9×	6e-04	CSF2RA, CSF2RB
Interleukin-3, Interleukin-5 and GM-CSF signaling	2	105.7×	8e-04	CSF2RA, CSF2RB
Defective ABCA3 causes SMDP3	1	1903.3×	0.002	ABCA3
Defective pro-SFTPB causes SMDP1 and RDS	1	1903.3×	0.002	SFTPB
Defective pro-SFTPC causes SMDP2 and RDS	1	1903.3×	0.002	SFTPC
Beta oxidation of myristoyl-CoA to lauroyl-CoA	1	634.4×	0.004	ACADL
Diseases associated with surfactant metabolism	1	475.8×	0.005	ABCA3
Beta oxidation of lauroyl-CoA to decanoyl-CoA-CoA	1	380.7×	0.006	ACADL
mitochondrial fatty acid beta-oxidation of unsaturated fatty acids	1	317.2×	0.007	ACADL
mitochondrial fatty acid beta-oxidation of saturated fatty acids	1	271.9×	0.007	ACADL
RAF/MAP kinase cascade	2	20.4×	0.007	CSF2RA, CSF2RB
ABC transporters in lipid homeostasis	1	100.2×	0.017	ABCA3
ABC transporter disorders	1	73.2×	0.022	ABCA3
Mitochondrial Fatty Acid Beta-Oxidation	1	63.4×	0.023	ACADL
Disorders of transmembrane transporters	1	23.2×	0.060	ABCA3
Fatty acid metabolism	1	21.9×	0.061	ACADL
ABC-family protein mediated transport	1	20.2×	0.062	ABCA3
Diseases of metabolism	1	13.4×	0.089	ABCA3
Metabolism of lipids	1	5.3×	0.206	ACADL
Transport of small molecules	1	4.2×	0.243	ABCA3
Disease	1	2.2×	0.410	ABCA3
Metabolism of proteins	1	2.1×	0.412	ABCA3
Metabolism	1	1.9×	0.417	ACADL

GO biological processes by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 6 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

GO term	Cohort genes	Fold	FDR	Sample cohort genes
respiratory gaseous exchange by respiratory system	3	312.1×	3e-06	SFTPB, SFTPC, CSF2RB
granulocyte-macrophage colony-stimulating factor signaling pathway	2	1404.3×	1e-05	CSF2RA, CSF2RB
positive regulation of leukocyte proliferation	2	936.2×	2e-05	CSF2RA, CSF2RB
cell surface receptor signaling pathway via JAK-STAT	2	96.8×	0.002	CSF2RA, CSF2RB
positive regulation of protein homooligomerization	1	2808.7×	0.003	ABCA3
carnitine catabolic process	1	2808.7×	0.003	ACADL
regulation of phosphatidylcholine metabolic process	1	1404.3×	0.005	ABCA3
carnitine metabolic process, CoA-linked	1	936.2×	0.005	ACADL
xenobiotic export from cell	1	936.2×	0.005	ABCA3
positive regulation of phospholipid efflux	1	702.2×	0.006	ABCA3
cellular response to interleukin-3	1	468.1×	0.006	CSF2RB
interleukin-5-mediated signaling pathway	1	468.1×	0.006	CSF2RB
long-chain fatty acid catabolic process	1	468.1×	0.006	ACADL
regulation of lipid biosynthetic process	1	468.1×	0.006	ABCA3
organelle assembly	1	468.1×	0.006	ABCA3
interleukin-3-mediated signaling pathway	1	401.2×	0.007	CSF2RB
positive regulation of phospholipid transport	1	401.2×	0.007	ABCA3
negative regulation of fatty acid oxidation	1	280.9×	0.009	ACADL
fatty acid beta-oxidation using acyl-CoA dehydrogenase	1	234.1×	0.010	ACADL
phosphatidylglycerol metabolic process	1	234.1×	0.010	ABCA3
growth hormone receptor signaling pathway	1	200.6×	0.011	CSF2RA
regulation of cholesterol metabolic process	1	187.2×	0.011	ACADL
sphingolipid metabolic process	1	165.2×	0.011	SFTPB
phospholipid homeostasis	1	165.2×	0.011	ABCA3
xenobiotic transmembrane transport	1	156.0×	0.012	ABCA3
negative regulation of fatty acid biosynthetic process	1	147.8×	0.012	ACADL
xenobiotic transport	1	140.4×	0.012	ABCA3
surfactant homeostasis	1	133.8×	0.012	ABCA3
phosphatidylcholine metabolic process	1	133.8×	0.012	ABCA3
phospholipid transport	1	117.0×	0.012	ABCA3

Therapeutics

Drug target analysis

Approved (phase 4): 0 · Phase ≥3: 0 · Phased (≥1): 0 · Undrugged: 6

Druggability breadth: 2 of 6 evidence-associated genes (33%) have a ChEMBL target (buckets above are over the deeply-mined display cohort).

Top cohort targets by molecule count

Symbol	Molecules	Max phase
CSF2RA	0	0
CSF2RB	0	0
ACADL	0	0
SFTPB	0	0
SFTPC	0	0
ABCA3	0	0

Bioactivity and enzyme data

Enzyme cohort genes (≥1 EC): 0.

Pharmacogenomics

Cohort genes with a PharmGKB record: 6; with CPIC/DPWG dosing guidelines: 0.

No cohort gene has a CPIC/DPWG genotype-guided dosing guideline (PharmGKB).

Chemical tractability of cohort targets

0 approved/phased compounds have measured bioactivity against a cohort gene (and aren’t yet in disease-level trials). This is a research / tractability signal, NOT a therapeutic recommendation — a bioactivity row often reflects off-target or screening binding (e.g. promiscuous kinase inhibitors against a cohort kinase), implying no disease mechanism.

Druggability pyramid

Cohort genes binned by druggability tier (high → low):

Tier	Definition	Genes	Symbols
A	Approved (phase 4 drug)	0
B	Phased (≥1) drug, not yet approved	0
C	Druggable family + PDB, no drug	3	CSF2RA, CSF2RB, ABCA3
D	Druggable family + AlphaFold only, no drug	0
E	Difficult family or no structure, no drug	3	ACADL, SFTPB, SFTPC

Undrugged target profiles

6 cohort genes are undrugged. Ranked by ‘starting-point quality’ (assay depth + drugged-partner adjacency).

Symbol	ChEMBL assays	Drugged partners (top 3)
CSF2RA	0	—
CSF2RB	0	—
ACADL	0	—
SFTPB	0	—
SFTPC	0	—
ABCA3	0	—

Clinical trials & evidence

Clinical trials

Clinical trials: 2.

Phase distribution (across all retrieved trials)

Phase	Trials
PHASE2	1
PHASE1/PHASE2	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT05761899	PHASE1/PHASE2	RECRUITING	Safety and Efficacy of PMT Therapy of hPAP
NCT01511068	PHASE2	COMPLETED	Inhaled Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) in Hereditary Pulmonary Alveolar Proteinosis (PAP)

Drugs tested across these trials (top 30)

Molecule	Max phase	Trials referencing
SARGRAMOSTIM	4	1