FTHL17

Summary

FTHL17 (ferritin heavy chain like 17, HGNC:3987) is a protein-coding gene on chromosome Xp21.2, encoding Ferritin heavy polypeptide-like 17 (Q9BXU8).

This gene encodes a ferritin heavy chain-like protein. This gene is primarily expressed in embryonic germ cells. The encoded protein may lack ferroxidase activity. Multiple pseudogenes of this gene are found on chromosome X.

Source: NCBI Gene 53940 — RefSeq curated summary.

At a glance

• GWAS associations: 1
• Clinical variants (ClinVar): 24 total
• MANE Select transcript: NM_031894

Identifiers

Gene identifiers

Field	Value
HGNC ID	HGNC:3987
Approved symbol	FTHL17
Name	ferritin heavy chain like 17
Location	Xp21.2
Locus type	gene with protein product
Status	Approved
Aliases	CT38
Ensembl gene	ENSG00000132446
Ensembl biotype	protein_coding
OMIM	300308
Entrez	53940

Gene structure

Transcript identifiers

Ensembl transcripts: 1 — 1 protein_coding

ENST00000359202

RefSeq mRNA: 1 — MANE Select: NM_031894 NM_031894

CCDS: CCDS14227

Canonical transcript exons

ENST00000359202 — 1 exons

Exon	Start	End
ENSE00001431309	31071233	31072041

Expression profiles

Bgee: expression breadth broad, 11 present calls, max score 80.51.

FANTOM5 (CAGE): breadth tissue_specific, TPM avg 0.0813 / max 121.0469, expressed in 5 samples.

FANTOM5 promoters (2 alternative TSS)

Promoter ID	TPM avg	Samples expressed
198794	0.0685	4
198793	0.0128	2

Top tissues by expression

244 total, by Bgee expression score (0-100, higher = more expressed):

Tissue	Anatomy ID	Expression score	Quality
male germ line stem cell (sensu Vertebrata) in testis	CL:0000089 ∩ UBERON:0000473	80.51	silver quality
oocyte	CL:0000023	71.15	silver quality
secondary oocyte	CL:0000655	68.33	gold quality
right testis	UBERON:0004534	63.39	gold quality
left testis	UBERON:0004533	62.46	gold quality
testis	UBERON:0000473	60.76	gold quality
lower lobe of lung	UBERON:0008949	51.70	silver quality
primordial germ cell in gonad	CL:0000670 ∩ UBERON:0000991	48.42	silver quality
adult organism	UBERON:0007023	48.19	gold quality
substantia nigra pars reticulata	UBERON:0001966	47.24	gold quality
substantia nigra pars compacta	UBERON:0001965	46.57	gold quality
skeletal muscle tissue of biceps brachii	UBERON:0004502	43.66	gold quality
skeletal muscle tissue of rectus abdominis	UBERON:0004511	43.37	gold quality
jejunum	UBERON:0002115	43.15	gold quality
biceps brachii	UBERON:0001507	42.25	gold quality
urethra	UBERON:0000057	42.13	gold quality
vastus lateralis	UBERON:0001379	41.69	gold quality
quadriceps femoris	UBERON:0001377	41.64	gold quality
superficial temporal artery	UBERON:0001614	41.33	gold quality
palpebral conjunctiva	UBERON:0001812	41.10	gold quality
mucosa of paranasal sinus	UBERON:0005030	40.98	gold quality
amniotic fluid	UBERON:0000173	40.69	gold quality
jejunal mucosa	UBERON:0000399	40.59	gold quality
bronchial epithelial cell	CL:0002328	40.49	gold quality
epithelium of nasopharynx	UBERON:0001951	40.45	gold quality
myocardium	UBERON:0002349	40.45	gold quality
bronchus	UBERON:0002185	40.44	gold quality
gingival epithelium	UBERON:0001949	40.43	gold quality
germinal epithelium of ovary	UBERON:0001304	40.33	gold quality
esophagus squamous epithelium	UBERON:0006920	40.29	gold quality

Single-cell (SCXA)

Detected in 1 experiment(s), a significant marker in 0.

Experiment	Marker?	Max mean expression
E-ANND-3	no	0.65

Regulation

Is transcription factor: no

miRNA regulators (miRDB)

14 targeting FTHL17, top 30 by miRDB confidence (max_score; target_count = how many genes the miRNA targets in total — lower means more specific):

miRNA	Max score	Avg score	miRNA target_count
HSA-MIR-9983-3P	99.94	71.48	3631
HSA-MIR-129-5P	99.88	70.26	3273
HSA-MIR-137-3P	99.87	74.74	2401
HSA-MIR-5582-3P	99.86	72.48	4221
HSA-MIR-548AR-3P	99.85	71.26	3889
HSA-MIR-548AZ-3P	99.82	70.56	3549
HSA-MIR-548BC	99.82	70.61	3524
HSA-MIR-548E-3P	99.82	70.59	3514
HSA-MIR-548F-3P	99.82	70.59	3540
HSA-MIR-4639-5P	99.81	67.37	1028
HSA-MIR-577	99.78	69.13	2479
HSA-MIR-548A-3P	99.76	70.58	3524
HSA-MIR-4766-5P	99.75	69.23	2662
HSA-MIR-589-5P	98.72	66.96	927

Literature-anchored findings (GeneRIF, showing 2)

• FTHL17 encodes a ferritin-like protein without ferroxidase activity. Its restricted embryonic expression and partial nuclear localization suggest that this novel ferritin type may have functions other than iron storage. (PMID:25749565)
• Describe the first 46,XY PGD pedigree that may be attributed to mutations of the FTHL17 gene and speculate that the FTHL17 gene is involved in the testis-determining pathway and tumorigenesis. (PMID:30922653)

Cross-species orthologs

5 orthologs

Organism	Symbol	Gene ID
danio_rerio	zgc:56095	ENSDARG00000018461
danio_rerio	zgc:172145	ENSDARG00000045586
drosophila_melanogaster	Fer3HCH	FBGN0030449
caenorhabditis_elegans		WBGENE00001500
caenorhabditis_elegans	ftn-2	WBGENE00001501

Paralogs (3): FTL (ENSG00000087086), FTH1 (ENSG00000167996), FTMT (ENSG00000181867)

Protein

Protein identifiers

Ferritin heavy polypeptide-like 17 — Q9BXU8 (reviewed: Q9BXU8)

Alternative names: Cancer/testis antigen 38

All UniProt accessions (2): Q9BXU8, A0A384NPV7

UniProt curated annotations — full annotation on UniProt →

Tissue specificity. Testis specific. Also expressed in several cancers.

Similarity. Belongs to the ferritin family.

RefSeq proteins (1): NP_114100* (*=MANE)

Domains & families (InterPro)

ID	Name	Type
IPR001519	Ferritin	Family
IPR008331	Ferritin_DPS_dom	Domain
IPR009040	Ferritin-like_diiron	Domain
IPR009078	Ferritin-like_SF	Homologous_superfamily
IPR012347	Ferritin-like	Homologous_superfamily
IPR014034	Ferritin_CS	Conserved_site

Pfam: PF00210

UniProt features (8 total): binding site 4, sequence variant 2, chain 1, domain 1

Structure

Experimental structures (PDB)

0 structures.

Predicted structure (AlphaFold)

Model	pLDDT	Fraction very-high
AF-Q9BXU8-F1	93.35	0.86

Functional residue map

Curated UniProt residues grouped by drug-discovery relevance — catalytic, ligand-binding, modification, and mutation-validated positions. Source: UniProtKB sequence features.

Ligand- & substrate-binding residues (4): 28; 66; 108; 142

Function

Pathways and Gene Ontology

Reactome pathways

0 pathways

MSigDB gene sets: 51 (showing top): CREL_01, GOBP_TRANSITION_METAL_ION_TRANSPORT, SP3_Q3, GOBP_INTRACELLULAR_IRON_ION_HOMEOSTASIS, GOBP_IRON_ION_TRANSPORT, GOBP_MONOATOMIC_CATION_TRANSPORT, NFKB_C, GGGNNTTTCC_NFKB_Q6_01, GOBP_MONOATOMIC_ION_HOMEOSTASIS, TGGAAA_NFAT_Q4_01, TAATTA_CHX10_01, GOBP_HOMEOSTATIC_PROCESS, GOBP_CHEMICAL_HOMEOSTASIS, GOMF_FERROUS_IRON_BINDING, GOMF_IRON_ION_BINDING

GO Biological Process (2): iron ion transport (GO:0006826), intracellular iron ion homeostasis (GO:0006879)

GO Molecular Function (4): ferrous iron binding (GO:0008198), ferric iron binding (GO:0008199), protein binding (GO:0005515), metal ion binding (GO:0046872)

GO Cellular Component (1): cytoplasm (GO:0005737)

GO top-level categories

Rollup of top GO terms by namespace:

Category	Terms
iron ion binding	2
transition metal ion transport	1
intracellular monoatomic cation homeostasis	1
inorganic ion homeostasis	1
binding	1
cation binding	1
intracellular anatomical structure	1
cellular anatomical structure	1

Protein interactions and networks

STRING

830 interactions, top by confidence (×1000):

Protein A	Protein B	Partner UniProt	Score
FTHL17	OR6X1	Q8NH79	541
FTHL17	AQP7B	A0A075B734	523
FTHL17	ZNF165	P49910	479
FTHL17	CT55	Q8WUE5	476
FTHL17	MAGEB2	O15479	476
FTHL17	SPANXN1	Q5VSR9	450
FTHL17	CSAG1	Q6PB30	448
FTHL17	XAGE1B	Q9HD64	430
FTHL17	CPXCR1	Q8N123	424
FTHL17	KRTAP19-7	Q3SYF9	419
FTHL17	OR13J1	Q8NGT2	417
FTHL17	GAGE4	P0DSO3	417
FTHL17	PRAME	P78395	414
FTHL17	GMCL2	Q8NEA9	414
FTHL17	MAGEB1	P43366	408

IntAct

19 interactions, top by confidence:

A	B	Type	Score
NOTCH2NLC	FTHL17	psi-mi:“MI:0915”(physical association)	0.560
TMEM239	FTHL17	psi-mi:“MI:0915”(physical association)	0.560
KRTAP10-8	FTHL17	psi-mi:“MI:0915”(physical association)	0.560
FTHL17	PUF60	psi-mi:“MI:0915”(physical association)	0.560
GSG1	FTHL17	psi-mi:“MI:0915”(physical association)	0.560
SLC35E1	FTHL17	psi-mi:“MI:0915”(physical association)	0.560
FTHL17	NOTCH2NLC	psi-mi:“MI:0915”(physical association)	0.000
FTHL17	TMEM239	psi-mi:“MI:0915”(physical association)	0.000
FTHL17	KRTAP10-8	psi-mi:“MI:0915”(physical association)	0.000
FTHL17	PUF60	psi-mi:“MI:0915”(physical association)	0.000
FTHL17	SLC35E1	psi-mi:“MI:0915”(physical association)	0.000
FTHL17	GSG1	psi-mi:“MI:0915”(physical association)	0.000

BioGRID (8): FTHL17 (Affinity Capture-MS), FTHL17 (Two-hybrid), FTHL17 (Two-hybrid), PUF60 (Two-hybrid), KRTAP10-8 (Two-hybrid), TMEM239 (Two-hybrid), NOTCH2NL (Two-hybrid), NBPF19 (Two-hybrid)

ESM2 similar proteins: O46119, O46414, O46415, P02791, P02792, P02793, P02794, P07229, P07797, P07798, P08267, P09451, P09528, P0A999, P0A9A1, P17663, P18685, P19130, P19132, P19133, P25319, P25320, P25915, P29389, P29391, P42577, P49946, P49947, P49948, P80145, P85835, P85836, P85837, P85838, P85839, Q26061, Q2MHN1, Q2MHN2, Q2YDI9, Q53VB8

Diamond homologs: E1WS50, O46414, P02794, P07229, P08267, P09528, P0A998, P0A999, P0A9A0, P0A9A1, P0CJ83, P17663, P18685, P19130, P19132, P25915, P29389, P43707, P43708, P49946, P49947, P49948, P52093, P80145, P85835, P85836, P85837, P85838, P85839, Q26061, Q2FFK2, Q2FWZ8, Q2MHN2, Q2YDI9, Q2YU34, Q46106, Q4L7K6, Q5HEN0, Q5R8J7, Q6G840

SIGNOR signaling

0 interactions.

Disease & clinical

Clinical variants and AI predictions

ClinVar

24 variants total. Per-class counts are floors (≥ shown; pagination cap):

Classification	Count (floor)
Pathogenic	0
Likely pathogenic	0
Uncertain significance	19
Likely benign	4
Benign	1

Top pathogenic / likely-pathogenic (0)

SpliceAI

108 predictions. Top by Δscore:

Variant	Effect	Δscore
X:31071692:T:TA	donor_gain	0.8800
X:31071743:T:C	donor_gain	0.5900
X:31071761:C:CA	donor_gain	0.5800
X:31071719:C:CT	donor_gain	0.5500
X:31071718:C:CT	donor_gain	0.5400
X:31071920:T:TA	donor_gain	0.5300
X:31071749:T:TA	donor_gain	0.5200
X:31071453:AG:A	donor_gain	0.5100
X:31071821:C:A	donor_gain	0.5000
X:31071454:G:C	donor_gain	0.4900
X:31071577:C:CA	donor_gain	0.4900
X:31071746:G:C	donor_gain	0.4900
X:31071550:T:A	donor_gain	0.4800
X:31071498:GTAGC:G	donor_gain	0.4700
X:31071772:T:TA	donor_gain	0.4700
X:31071848:G:C	donor_gain	0.4700
X:31071524:TG:T	donor_gain	0.4500
X:31071679:TGGC:T	donor_gain	0.4500
X:31071693:C:A	donor_gain	0.4500
X:31071840:C:CT	acceptor_gain	0.4500
X:31071918:CTT:C	donor_gain	0.4400
X:31071499:TAGCC:T	donor_gain	0.4300
X:31071500:AGCCA:A	donor_gain	0.4300
X:31071824:G:T	acceptor_gain	0.4300
X:31071917:A:AC	donor_gain	0.4300
X:31071918:C:CC	donor_gain	0.4300
X:31071674:AG:A	donor_gain	0.4200
X:31071820:T:TA	donor_gain	0.4100
X:31071626:T:A	donor_gain	0.4000
X:31071736:AG:A	donor_gain	0.4000

AlphaMissense

1206 scored. Top likely-pathogenic:

Variant	Protein change	am_pathogenicity
X:31071828:G:C	F42L	0.898
X:31071828:G:T	F42L	0.898
X:31071830:A:G	F42L	0.898
X:31071441:G:C	F171L	0.885
X:31071441:G:T	F171L	0.885
X:31071443:A:G	F171L	0.885
X:31071786:G:C	F56L	0.883
X:31071786:G:T	F56L	0.883
X:31071788:A:G	F56L	0.883
X:31071555:G:C	F133L	0.878
X:31071555:G:T	F133L	0.878
X:31071557:A:G	F133L	0.878
X:31071834:G:C	F40L	0.859
X:31071834:G:T	F40L	0.859
X:31071836:A:G	F40L	0.859
X:31071725:G:T	R77S	0.805
X:31071823:C:G	R44P	0.790
X:31071672:C:A	W94C	0.788
X:31071672:C:G	W94C	0.788
X:31071674:A:G	W94R	0.779
X:31071674:A:T	W94R	0.779
X:31071655:G:T	A100D	0.774
X:31071610:A:G	L115P	0.770
X:31071798:G:C	F52L	0.767
X:31071798:G:T	F52L	0.767
X:31071800:A:G	F52L	0.767
X:31071644:C:G	A104P	0.764
X:31071601:A:G	L118P	0.761
X:31071724:C:G	R77P	0.761
X:31071589:G:T	A122D	0.756

dbSNP variants (sampled 300 via entrez): RS1001602359 (X:31072271 C>T), RS1003461151 (X:31072765 A>G,T), RS1003535219 (X:31071161 A>G), RS1004080251 (X:31071418 C>T), RS1005746557 (X:31073031 G>T), RS1008080914 (X:31071385 G>C,T), RS1010770945 (X:31072273 C>A), RS1013308539 (X:31072967 G>A), RS1014111992 (X:31072257 C>T), RS1015651784 (X:31073523 G>T), RS1015767767 (X:31073044 A>C), RS1016480895 (X:31072159 C>A), RS1019133678 (X:31072686 A>T), RS1020795330 (X:31072275 C>G,T), RS1021577336 (X:31071906 G>C)

Disease associations

OMIM: gene MIM:300308 | disease phenotypes: MIM:310200

GenCC curated gene-disease

Mondo (1): Duchenne muscular dystrophy (MONDO:0010679)

Orphanet (1): Duchenne muscular dystrophy (Orphanet:98896)

HPO phenotypes

0 total (0 of 0 shown, HPO-id order):

GWAS associations

1 associations (top):

Study	Trait	p-value
GCST009391_629	Metabolite levels	2.000000e-07

EFO canonical traits (1, from GWAS)

EFO ID	Trait name
EFO:0010503	inosine measurement

MeSH disease descriptors (1)

Descriptor	Name	Tree numbers
D020388	Muscular Dystrophy, Duchenne	C05.651.534.500.300; C10.668.491.175.500.300; C16.320.322.562; C16.320.577.300

Drugs & pharmacology

Drug and pharmacology data

Is drug target: no

PharmGKB: 1 entry (VIP=true, CPIC=false)

CTD chemical–gene interactions

16 total (human), top 16 by PubMed support.

Chemical	Actions (top 5)	PubMed papers
Valproic Acid	affects cotreatment, increases expression, increases methylation	2
tris(1,3-dichloro-2-propyl)phosphate	increases expression	1
sodium arsenite	increases expression	1
aflatoxin B2	increases methylation	1
CGP 52608	affects binding, increases reaction	1
K 7174	increases expression	1
Grape Seed Proanthocyanidins	affects cotreatment, increases expression	1
Resveratrol	affects cotreatment, decreases expression	1
Benzo(a)pyrene	affects methylation, decreases methylation	1
Catechin	affects cotreatment, increases expression	1
Diethylhexyl Phthalate	increases expression	1
Hydralazine	affects cotreatment, increases expression	1
Plant Extracts	affects cotreatment, decreases expression	1
Tobacco Smoke Pollution	increases expression	1
Aflatoxin B1	increases methylation	1
Copper Sulfate	increases expression	1

Clinical trials (associated diseases)

300 trials via MONDO — disease-level, not drug-specific.

Trial	Phase	Status	Title
NCT00606775	PHASE4	UNKNOWN	The Preventive Efficacy of Carvedilol on Cardiac Dysfunction in Duchenne Muscular Dystrophy
NCT00819845	PHASE4	UNKNOWN	Ramipril Versus Carvedilol in Duchenne and Becker Patients
NCT01422200	PHASE4	COMPLETED	Flu Vaccine Study in Neuromuscular Patients 2011
NCT01999075	PHASE4	COMPLETED	Stacking Exercises Aid the Decline in FVC and Sick Time
NCT04687020	PHASE4	ACTIVE_NOT_RECRUITING	Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502)
NCT04708314	PHASE4	TERMINATED	An Open-Label Study of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy
NCT05412394	PHASE4	RECRUITING	Once Weekly Infant Corticosteroid Trial for DMD
NCT06713135	PHASE4	ACTIVE_NOT_RECRUITING	A Study on Safety and Effectiveness of Long-term Treatment With Vamorolone in Boys With Duchenne Muscular Dystrophy
NCT07542314	PHASE4	NOT_YET_RECRUITING	Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting
NCT00004646	PHASE3	COMPLETED	Phase III Randomized, Double-Blind Study of Prednisone for Duchenne Muscular Dystrophy
NCT00110669	PHASE3	COMPLETED	High-dose Prednisone in Duchenne Muscular Dystrophy
NCT00308113	PHASE3	TERMINATED	CoQ10 and Prednisone in Non-Ambulatory DMD
NCT00839033	PHASE3	TERMINATED	Evaluation of a Mechanical Device During Acute Respiratory Failure in Patients With Neuromuscular Disorders
NCT01247207	PHASE3	COMPLETED	Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)
NCT01557400	PHASE3	COMPLETED	Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada
NCT01603407	PHASE3	COMPLETED	Finding the Optimum Regimen for Duchenne Muscular Dystrophy
NCT01648634	PHASE3	COMPLETED	Nebivolol for the Prevention of Left Ventricular Systolic Dysfunction in Patients With Duchenne Muscular Dystrophy
NCT02255552	PHASE3	COMPLETED	Study of Eteplirsen in DMD Patients
NCT02354352	PHASE3	COMPLETED	Therapeutic Potential for Aldosterone Inhibition in Duchenne Muscular Dystrophy
NCT02500381	PHASE3	COMPLETED	Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)
NCT02814019	PHASE3	TERMINATED	A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids
NCT02851797	PHASE3	COMPLETED	Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy
NCT03354039	PHASE3	COMPLETED	Tamoxifen in Duchenne Muscular Dystrophy
NCT03532542	PHASE3	TERMINATED	An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy
NCT03603288	PHASE3	TERMINATED	Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E)
NCT03642145	PHASE3	WITHDRAWN	A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)
NCT03917719	PHASE3	TERMINATED	An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy
NCT04060199	PHASE3	COMPLETED	Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)
NCT04281485	PHASE3	ACTIVE_NOT_RECRUITING	Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy
NCT04371666	PHASE3	TERMINATED	Phase 3 Trial of Pamrevlumab or Placebo With Systemic Corticosteroids in Participants With Non-ambulatory Duchenne Muscular Dystrophy (DMD)
NCT04587908	PHASE3	ACTIVE_NOT_RECRUITING	A Phase 3 Study of TAS-205 in Patients With Duchenne Muscular Dystrophy（REACH-DMD）
NCT04632940	PHASE3	TERMINATED	Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids in Participants With Ambulatory DMD
NCT04768062	PHASE3	UNKNOWN	Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X)
NCT05096221	PHASE3	COMPLETED	A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
NCT05689164	PHASE3	TERMINATED	A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy.
NCT05881408	PHASE3	ACTIVE_NOT_RECRUITING	A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)
NCT05933057	PHASE3	RECRUITING	Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy
NCT05967351	PHASE3	ENROLLING_BY_INVITATION	A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study
NCT07160634	PHASE3	RECRUITING	A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)
NCT07587242	PHASE3	NOT_YET_RECRUITING	A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping