Becker muscular dystrophy

Summary

Becker muscular dystrophy (MONDO:0010311) is a disease caused by DMD (GenCC Definitive), with 4 cohort genes and 72 clinical trials. Top therapeutic interventions include ataluren, lisinopril anhydrous, and carvedilol.

At a glance

• Prevalence: 1-9 / 100 000 (Europe) [Orphanet-validated]
• Causal gene: DMD (GenCC Definitive)
• Cohort genes: 4
• ClinVar variants: 1,526
• Phenotypes (HPO): 16
• Clinical trials: 72

Clinical features

Epidemiology

Prevalence records

13 prevalence record(s), Orphanet:

Type	Class	Value	Geography	Validation
Point prevalence	1-9 / 100 000	2	Europe	Validated
Prevalence at birth	1-9 / 100 000	2.2	Europe	Validated
Point prevalence	1-9 / 100 000	3.64	United Kingdom	Validated
Point prevalence	1-9 / 100 000	2.47	Italy	Validated
Point prevalence	1-9 / 100 000	3.94	Egypt	Validated
Point prevalence	1-9 / 1 000 000	0.91	Japan	Validated
Point prevalence	<1 / 1 000 000	0.07	South Africa	Validated
Point prevalence	1-9 / 100 000	1.59	Ireland	Validated
Point prevalence	1-9 / 100 000	1.42	Puerto rico	Validated
Prevalence at birth	1-9 / 100 000	2.7	United Kingdom	Validated
Prevalence at birth	1-9 / 100 000	2.5	United States	Validated
Point prevalence	1-9 / 100 000	1.53	Worldwide	Not yet validated
Point prevalence	1-9 / 100 000	2.4	United States	Not yet validated

Signs & symptoms

Clinical features (HPO)

16 HPO clinical features (Orphanet curated; top 16 by frequency):

HPO ID	Term	Frequency
HP:0001288	Gait disturbance	Very frequent (80-99%)
HP:0002913	Myoglobinuria	Very frequent (80-99%)
HP:0003236	Elevated circulating creatine kinase concentration	Very frequent (80-99%)
HP:0003326	Myalgia	Very frequent (80-99%)
HP:0003546	Exercise intolerance	Very frequent (80-99%)
HP:0003551	Difficulty climbing stairs	Very frequent (80-99%)
HP:0012086	Abnormal urinary color	Very frequent (80-99%)
HP:0001324	Muscle weakness	Frequent (30-79%)
HP:0002527	Falls	Frequent (30-79%)
HP:0002814	Abnormality of the lower limb	Frequent (30-79%)
HP:0002910	Elevated circulating hepatic transaminase concentration	Frequent (30-79%)
HP:0003394	Muscle spasm	Frequent (30-79%)
HP:0012378	Fatigue	Frequent (30-79%)
HP:0001763	Pes planus	Occasional (5-29%)
HP:0003202	Skeletal muscle atrophy	Occasional (5-29%)
HP:0030051	Tip-toe gait	Occasional (5-29%)

Identifiers

Disease identifiers

Field	Value
Canonical name	Becker muscular dystrophy
Mondo ID	MONDO:0010311
MeSH	C570377
OMIM	300376
Orphanet	98895
DOID	DOID:9883
ICD-11	690532643
NCIT	C84587
SNOMED CT	387732009
UMLS	C0917713
MedGen	182959
GARD	0005900
MedDRA	10059117
Is cancer (heuristic)	no

Also known as: Becker dystrophinopathy · Becker muscular dystrophy · Becker muscular dystrophy, X-linked recessive · Becker’s muscular dystrophy · BMD · muscular dystrophy pseudohypertrophic progressive, Becker type · muscular dystrophy, Becker type

Data availability: 1,526 ClinVar variants · 2 GenCC gene-disease records · 26 cell lines.

Disease family

An umbrella term covering 1 Mondo subtype.

Classification path: disease › human disease › disease by body system or component › musculoskeletal system disorder › muscle tissue disorder › skeletal muscle disorder › myopathy › muscular dystrophy › DMD-related muscular dystrophy › Becker muscular dystrophy

Related subtypes (1): Duchenne muscular dystrophy

Subtypes (1): muscular dystrophy, pseudohypertrophic, with Internalized capillaries

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:

158 uncertain significance, 136 conflicting classifications of pathogenicity, 105 pathogenic, 72 likely benign, 45 benign/likely benign, 36 likely pathogenic, 30 benign, 18 pathogenic/likely pathogenic

ClinVar	Variant (HGVS)	Gene	Classification	Review
1327590	Multiple alleles		Pathogenic	criteria provided, single submitter
1071032	NM_004006.3(DMD):c.568C>T (p.Gln190Ter)	DMD	Pathogenic	criteria provided, single submitter
1071901	NM_004006.3(DMD):c.8416C>T (p.Gln2806Ter)	DMD	Pathogenic	criteria provided, multiple submitters, no conflicts
1073643	NM_004006.3(DMD):c.10224dup (p.Pro3409fs)	DMD	Pathogenic	criteria provided, multiple submitters, no conflicts
1076188	NM_004006.3(DMD):c.574_577del (p.Ala192fs)	DMD	Pathogenic	criteria provided, multiple submitters, no conflicts
11211	NM_004006.3(DMD):c.8944C>T (p.Arg2982Ter)	DMD	Pathogenic	criteria provided, multiple submitters, no conflicts
11213	NM_004006.3(DMD):c.10108C>T (p.Arg3370Ter)	DMD	Pathogenic	criteria provided, multiple submitters, no conflicts
11225	NM_004006.3(DMD):c.433C>T (p.Arg145Ter)	DMD	Pathogenic	criteria provided, multiple submitters, no conflicts
11226	NM_004006.3(DMD):c.2380+3A>C	DMD	Pathogenic	no assertion criteria provided
11227	NM_004006.3(DMD):c.8548-1G>C	DMD	Pathogenic	no assertion criteria provided
11239	NM_004006.3(DMD):c.178C>T (p.Gln60Ter)	DMD	Pathogenic	criteria provided, multiple submitters, no conflicts
11241	NM_004006.3(DMD):c.691T>A (p.Tyr231Asn)	DMD	Pathogenic	no assertion criteria provided
11248	NM_004006.3(DMD):c.1602G>T (p.Lys534Asn)	DMD	Pathogenic	criteria provided, single submitter
11277	NM_004006.3(DMD):c.10477del (p.Gln3493fs)	DMD	Pathogenic	no assertion criteria provided
11280	NM_004006.3(DMD):c.3631G>T (p.Glu1211Ter)	DMD	Pathogenic	no assertion criteria provided
11283	NM_004006.3(DMD):c.3940C>T (p.Arg1314Ter)	DMD	Pathogenic	criteria provided, multiple submitters, no conflicts
11286	NM_004006.3(DMD):c.9225-285A>G	DMD	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
11288	NM_004006.3(DMD):c.8713C>T (p.Arg2905Ter)	DMD	Pathogenic	criteria provided, multiple submitters, no conflicts
1180800	NM_004006.3(DMD):c.6576G>A (p.Trp2192Ter)	DMD	Pathogenic	criteria provided, multiple submitters, no conflicts
1299643	NM_004006.3(DMD):c.71G>A (p.Trp24Ter)	DMD	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
1319977	NM_004006.3(DMD):c.5632C>T (p.Gln1878Ter)	DMD	Pathogenic	criteria provided, single submitter
1322237	NM_004006.3(DMD):c.2873C>G (p.Ser958Ter)	DMD	Pathogenic	criteria provided, single submitter
1322243	NM_004006.3(DMD):c.9249G>A (p.Trp3083Ter)	DMD	Pathogenic	criteria provided, multiple submitters, no conflicts
1322249	NM_004006.3(DMD):c.9109C>T (p.Gln3037Ter)	DMD	Pathogenic	criteria provided, single submitter
1322265	NM_004006.3(DMD):c.3556G>T (p.Glu1186Ter)	DMD	Pathogenic	criteria provided, multiple submitters, no conflicts
1322300	NM_004006.3(DMD):c.6973C>T (p.Gln2325Ter)	DMD	Pathogenic	criteria provided, single submitter
1322307	NM_004006.3(DMD):c.7750C>T (p.Gln2584Ter)	DMD	Pathogenic	criteria provided, single submitter
1322340	NM_004006.3(DMD):c.1375G>T (p.Glu459Ter)	DMD	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
1322358	NM_004006.3(DMD):c.2365G>T (p.Glu789Ter)	DMD	Pathogenic	criteria provided, single submitter
1322359	NM_004006.3(DMD):c.2669T>G (p.Leu890Ter)	DMD	Pathogenic	criteria provided, multiple submitters, no conflicts

Genes & proteins

Mendelian disease overlap and somatic drivers

GenCC: 12 · Orphanet: 11 · 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
DMD	Definitive	X-linked	Becker muscular dystrophy	12

Orphanet rare-disease linkage (cohort genes)

Gene	Orphanet ID	Rare disease
DMD	Orphanet:154	Familial isolated dilated cardiomyopathy
DMD	Orphanet:206546	Symptomatic form of muscular dystrophy of Duchenne and Becker in female carriers
DMD	Orphanet:777	X-linked non-syndromic intellectual disability
DMD	Orphanet:98895	Becker muscular dystrophy
DMD	Orphanet:98896	Duchenne muscular dystrophy
SNTA1	Orphanet:101016	Romano-Ward syndrome
PKP2	Orphanet:130	Brugada syndrome
PKP2	Orphanet:293888	Inherited isolated arrhythmogenic cardiomyopathy, dominant-left variant
PKP2	Orphanet:293899	Inherited isolated arrhythmogenic ventricular dysplasia, biventricular variant
PKP2	Orphanet:293910	Inherited isolated arrhythmogenic cardiomyopathy, dominant-right variant
PKP2	Orphanet:54260	Left ventricular noncompaction

Cohort genes → proteins

4 cohort genes, 4 distinct canonical proteins.

Evidence partition

Subset	Genes
multi_evidence	4

Cohort genes (full)

Symbol	HGNC	Ensembl	UniProt	Name	Evidence
DMD	HGNC:2928	ENSG00000198947	P11532	Dystrophin	gencc,clinvar
SNTA1	HGNC:11167	ENSG00000101400	Q13424	Alpha-1-syntrophin	clinvar
DDX53	HGNC:20083	ENSG00000184735	Q86TM3	Probable ATP-dependent RNA helicase DDX53	clinvar
PKP2	HGNC:9024	ENSG00000057294	Q99959	Plakophilin-2	clinvar

Cohort function summary

Lead sentence per gene, UniProt-curated.

Symbol	Protein name	Function (lead sentence)
DMD	Dystrophin	Anchors the extracellular matrix to the cytoskeleton via F-actin.
SNTA1	Alpha-1-syntrophin	Adapter protein that binds to and probably organizes the subcellular localization of a variety of membrane proteins.
PKP2	Plakophilin-2	A component of desmosome cell-cell junctions which are required for positive regulation of cellular adhesion.

Protein-family classification

Druggable: 0 · Difficult: 2 · Unknown: 2 · Druggable fraction: 0.0

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
Scaffold/PPI	1	4.3×	0.605
Transcription factor	1	2.1×	0.605
Other/Unknown	2	0.9×	0.769

Per-gene assignment

Symbol	Family	Druggable?	EC	InterPro (top 3)
DMD	Transcription factor	no		Znf_ZZ, WW_dom, Actinin_actin-bd_CS
SNTA1	Scaffold/PPI	no		PDZ, PH_domain, PH-like_dom_sf
DDX53	Other/Unknown	no		RNA-helicase_DEAD-box_CS, Helicase_C-like, KH_dom
PKP2	Other/Unknown	no		Armadillo, ARM-like, ARM-type_fold

Expression context

Cohort genes with no expression data: 0.

4 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)	1
broad (>20)	3
unknown	0

Top tissues across cohort

Tissue	Cohort genes
apex of heart	2
dorsal root ganglion	1
skeletal muscle tissue of rectus abdominis	1
trigeminal ganglion	1
gastrocnemius	1
hindlimb stylopod muscle	1
male germ line stem cell (sensu Vertebrata) in testis	1
primordial germ cell in gonad	1
sperm	1
heart right ventricle	1
left ventricle myocardium	1

Per-gene tissue summary (top 30)

Symbol	Bgee breadth	FANTOM5 breadth	SCXA	Top tissues
DMD	295	ubiquitous	marker	trigeminal ganglion, skeletal muscle tissue of rectus abdominis, dorsal root ganglion
SNTA1	266	ubiquitous	marker	apex of heart, hindlimb stylopod muscle, gastrocnemius
DDX53	12	tissue_specific	marker	primordial germ cell in gonad, male germ line stem cell (sensu Vertebrata) in testis, sperm
PKP2	237	ubiquitous	marker	heart right ventricle, apex of heart, left ventricle myocardium

Protein interactions among cohort

Intra-cohort edges: 1.

Hub genes (top 10 by interactor count)

Symbol	Interactor count
DDX53	2,965
DMD	2,479
PKP2	1,861
SNTA1	1,499

Intra-cohort edges

A	B	Sources
DMD	SNTA1	biogrid_interaction, intact, string_interaction

Structural data

PDB: 3 · AlphaFold-only: 1 · No structure: 0

Cohort genes with PDB structures (top 30)

Symbol	UniProt	PDB entries
DMD	P11532	6
DDX53	Q86TM3	2
PKP2	Q99959	1

AlphaFold-only cohort genes (top 30 by pLDDT)

Symbol	UniProt	pLDDT
SNTA1	Q13424	80.00

Function

Pathway analysis

Distinct Reactome pathways touched by cohort: 6. Enrichment computed across 4 evidence-associated genes (3 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 3 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

Pathway	Cohort genes	Fold	FDR	Sample cohort genes
Formation of the dystrophin-glycoprotein complex (DGC)	2	205.8×	2e-04	DMD, SNTA1
Non-integrin membrane-ECM interactions	2	102.9×	4e-04	DMD, SNTA1
Striated Muscle Contraction	1	102.9×	0.019	DMD
Formation of the cornified envelope	1	29.3×	0.051	PKP2
Extracellular matrix organization	1	21.0×	0.053	SNTA1
Keratinization	1	18.6×	0.053	PKP2

GO biological processes by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 3 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

GO term	Cohort genes	Fold	FDR	Sample cohort genes
regulation of sodium ion transmembrane transport	2	702.2×	7e-05	DMD, SNTA1
ventricular cardiac muscle cell action potential	2	660.9×	7e-05	SNTA1, PKP2
regulation of heart rate	2	312.1×	2e-04	DMD, SNTA1
regulation of muscle system process	1	5617.3×	0.001	DMD
regulation of cellular response to growth factor stimulus	1	5617.3×	0.001	DMD
maintenance of protein localization at cell tip	1	5617.3×	0.001	PKP2
cardiac muscle cell action potential	1	2808.7×	0.002	DMD
regulation of cell-substrate adhesion	1	1872.4×	0.003	PKP2
regulation of skeletal muscle contraction by regulation of release of sequestered calcium ion	1	1404.3×	0.003	DMD
peptide biosynthetic process	1	1404.3×	0.003	DMD
maintenance of animal organ identity	1	1123.5×	0.003	PKP2
regulation of substrate adhesion-dependent cell spreading	1	1123.5×	0.003	PKP2
regulation of skeletal muscle contraction	1	936.2×	0.004	DMD
intermediate filament bundle assembly	1	936.2×	0.004	PKP2
desmosome assembly	1	802.5×	0.004	PKP2
bundle of His cell-Purkinje myocyte adhesion involved in cell communication	1	802.5×	0.004	PKP2
desmosome organization	1	702.2×	0.004	PKP2
regulation of calcium ion transmembrane transport	1	702.2×	0.004	DMD
synaptic signaling	1	510.7×	0.005	DMD
cell communication by electrical coupling involved in cardiac conduction	1	468.1×	0.005	PKP2
regulation of ventricular cardiac muscle cell action potential	1	468.1×	0.005	PKP2
muscle cell development	1	312.1×	0.007	DMD
positive regulation of sodium ion transport	1	280.9×	0.007	PKP2
regulation of ventricular cardiac muscle cell membrane repolarization	1	280.9×	0.007	SNTA1
response to muscle stretch	1	255.3×	0.007	DMD
ventricular cardiac muscle tissue morphogenesis	1	234.1×	0.007	PKP2
cardiac muscle cell action potential involved in contraction	1	234.1×	0.007	PKP2
regulation of release of sequestered calcium ion into cytosol by sarcoplasmic reticulum	1	224.7×	0.007	DMD
regulation of cardiac muscle contraction by regulation of the release of sequestered calcium ion	1	224.7×	0.007	DMD
positive regulation of Rac protein signal transduction	1	216.1×	0.007	SNTA1

Therapeutics

Drug target analysis

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

Druggability breadth: 1 of 4 evidence-associated genes (25%) have a ChEMBL target (buckets above are over the deeply-mined display cohort).

Top cohort targets by molecule count

Symbol	Molecules	Max phase
DMD	0	0
SNTA1	0	0
DDX53	0	0
PKP2	0	0

Bioactivity and enzyme data

Enzyme cohort genes (≥1 EC): 0.

Pharmacogenomics

Cohort genes with a PharmGKB record: 4; 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	0
D	Druggable family + AlphaFold only, no drug	0
E	Difficult family or no structure, no drug	4	DMD, SNTA1, DDX53, PKP2

Undrugged target profiles

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

Symbol	ChEMBL assays	Drugged partners (top 3)
DMD	0	—
SNTA1	0	—
DDX53	0	—
PKP2	0	—

Clinical trials & evidence

Clinical trials

Clinical trials: 72.

Phase distribution (across all retrieved trials)

Phase	Trials
Not specified	41
PHASE2	13
PHASE1	9
PHASE4	3
PHASE2/PHASE3	2
PHASE3	2
PHASE1/PHASE2	2

Top trials by phase / activity

NCT	Phase	Status	Title
NCT00819845	PHASE4	UNKNOWN	Ramipril Versus Carvedilol in Duchenne and Becker Patients
NCT01070511	PHASE4	COMPLETED	Tadalafil in Becker Muscular Dystrophy
NCT05704088	PHASE4	COMPLETED	SGLT2 Inhibitors Between Reno Protective Effects and Impact on Bone and Mineral Disease Among Lupus Nephritis Patients
NCT01126697	PHASE2/PHASE3	COMPLETED	Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies
NCT01557400	PHASE3	COMPLETED	Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada
NCT02147639	PHASE2/PHASE3	COMPLETED	Effects of Sodium Nitrate on Blood Flow in Becker Muscular Dystrophy
NCT05457530	PHASE3	WITHDRAWN	Doravirine and Weight Gain in Antiretroviral Naive
NCT05291091	PHASE2	ACTIVE_NOT_RECRUITING	Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy (GRAND CANYON)
NCT06066580	PHASE2	ENROLLING_BY_INVITATION	Open-Label Extension of EDG-5506 in Participants With Becker Muscular Dystrophy
NCT00104078	PHASE1/PHASE2	COMPLETED	Study Evaluating MYO-029 in Adult Muscular Dystrophy
NCT00592553	PHASE2	COMPLETED	Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
NCT00847379	PHASE2	TERMINATED	Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
NCT01009294	PHASE2	TERMINATED	Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)
NCT01168908	PHASE2	TERMINATED	Revatio for Heart Disease in Duchenne Muscular Dystrophy and Becker Muscular Dystrophy
NCT01350154	PHASE2	COMPLETED	Effect of Modulating the nNOS System on Cardiac, Muscular and Cognitive Function in Becker Muscular Dystrophy Patients
NCT01540604	PHASE2	COMPLETED	CRD007 for the Treatment of Duchenne Muscular Dystrophy, Becker Muscular Dystrophy and Symptomatic Carriers
NCT01856868	PHASE1/PHASE2	COMPLETED	Use of (-)-Epicatechin in the Treatment of Becker Muscular Dystrophy (Pilot Study)
NCT02018731	PHASE2	COMPLETED	L-citrulline and Metformin in Becker’s Muscular Dystrophy
NCT03236662	PHASE2	COMPLETED	(-)- Epicatechin Becker Muscular Dystrophy
NCT03238235	PHASE2	COMPLETED	Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Becker Muscular Dystrophy
NCT04054375	PHASE2	COMPLETED	Weekly Steroids in Muscular Dystrophy
NCT05166109	PHASE2	COMPLETED	A Study to Assess Vamorolone in Becker Muscular Dystrophy (BMD)
NCT00005574	PHASE1	COMPLETED	Gentamicin Treatment of Muscular Dystrophy
NCT00873782	PHASE1	COMPLETED	Safety Study of Transvenous Limb Perfusion in Human Muscular Dystrophy
NCT01388764	PHASE1	COMPLETED	Safety, Tolerability and Effects of L-Arginine in Boys With Dystrophinopathy on Corticosteroids
NCT01519349	PHASE1	COMPLETED	Follistatin Gene Transfer to Patients With Becker Muscular Dystrophy and Sporadic Inclusion Body Myositis
NCT02434627	PHASE1	COMPLETED	Sodium Nitrate for Muscular Dystrophy
NCT02847975	PHASE1	COMPLETED	Sodium Nitrate to Improve Blood Flow
NCT04386304	PHASE1	COMPLETED	Safety and Biomarker Response to (+)-Epicatechin in Becker Muscular Dystrophy
NCT04585464	PHASE1	COMPLETED	A Study to Assess Safety, Tolerability, and PK of EDG-5506 in Healthy Volunteers and Becker Muscular Dystrophy Adults
NCT05160415	PHASE1	COMPLETED	A Study of EDG-5506 in Adult Males With Becker Muscular Dystrophy
NCT00390104	Not specified	RECRUITING	Molecular Analysis of Patients With Neuromuscular Disease
NCT01484678	Not specified	RECRUITING	Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy
NCT02069756	Not specified	RECRUITING	The Duchenne Registry
NCT02972580	Not specified	ACTIVE_NOT_RECRUITING	Characterization of Clinical Skeletal and Cardiac Impairment in Carriers of DMD and BMD
NCT04972604	Not specified	RECRUITING	CureDuchenne Link®: A Resource for Research
NCT05019625	Not specified	RECRUITING	Biomarker Development for Muscular Dystrophies
NCT05102916	Not specified	RECRUITING	Swiss Registry for Neuromuscular Disorders
NCT05257473	Not specified	ACTIVE_NOT_RECRUITING	Defining Endpoints in Becker Muscular Dystrophy
NCT05715957	Not specified	ENROLLING_BY_INVITATION	Follow-up Study on Female Carriers With DMD Gene Variants

Drugs tested across these trials (top 30)

Molecule	Max phase	Trials referencing
ATALUREN	4	4
LISINOPRIL ANHYDROUS	4	2
CARVEDILOL	4	1
DAPAGLIFLOZIN	4	1
GENTAMICIN	4	1
GIVINOSTAT	4	1
RAMIPRIL	4	1
TADALAFIL	4	1
VAMOROLONE	4	1
ARGININE	3	1
L-CITRULLINE	3	1
UBIDECARENONE	3	1
SEVASEMTEN	2	4
SODIUM NITRATE	2	3
(-)-EPICATECHIN	2	2
(+)-EPICATECHIN	2	1
STAMULUMAB	2	1
CHEMBL139521	0	1
CHEMBL177542	0	1
CHEMBL4244897	0	1
CHEMBL4281478	0	1
CHEMBL195892	0	1
CHEMBL3039597	0	1
(R)-Carvedilol	0	1
GENTAMICIN C1A	0	1
GENTAMICIN C2	0	1