6MWT	6-Minute Walk Test
AAV	Adeno-associated virus
ACTB	Actin beta
AD	Alzheimer’s disease
ADM	Abductor digiti minimi
Aldh4a1	Aldehyde dehydrogenase 4 family, member A1
ADMA	Asymmetric dimethylarginine
ALS	Amyotrophic lateral sclerosis
ALSFRS-R	Amyotrophic Lateral Sclerosis Functional Rating Scale—Revised
APP	Amyloid precursor protein
ASC-EVs	Exosomes derived from adipose-derived stem cells
ASOs	Antisense oligonucleotides
Aβ	Amyloid-β
BACE1	β-site APP cleaving enzyme-1
BBB	Blood–brain barrier
Bcl2	B-cell lymphoma 2
BI	Barthe index
BMI	Body mass index
Bmp4	Barthel Index
CCL5	C-C motif chemokine ligand 5
CCR	Creatinine-to-cystatin C ratio
ceRNA	Competing endogenous RNA
CHD4	Chromodomain helicase DNA binding protein 4
CHI3L1	Chitinase-3-like protein 1
CHIT1	Chitotriosidase 1
Chl1	Cell adhesion molecule L1-like protein
CHOP INTEND	Children’s Hospital of Philadelphia Infant test of Neuromuscular Disorders
CK	Creatine kinase
CMAP	Compound muscle action potential
CNS	Central nervous system
Cplx2	Complexin 2
Crn	Creatinine
CSF	Cerebrospinal fluid
DYNC1H1	Dynein cytoplasmic 1 heavy chain 1
EI	Muscle echogenicity
EIM	Electrical impedance myography
ELISA	Enzyme-linked immunosorbent assay
EMG	Electromyographic
Gata6	GATA binding protein 6
GDF15	Growth differentiation factor 15
GFAP	Glial fibrillary acidic protein
GSEA	Gene set enrichment analysis
HDL	High-density lipoprotein
HFMSE	Hammersmith functional motor scale–expanded
HINE-2	Section 2 of the Hammersmith Infant Neurological Examination
HNRNPA1	Heterogeneous nuclear ribonucleoprotein A1
HSPA7	Heat shock protein family A member 7
IBA1	Ionized calcium binding adaptor molecule 1
IL	Interleukin
IL-1ra	Interleukin-1 receptor antagonist
INF-γ	Interferon-γ
ISL1	ISL LIM homeobox 1
JUND	JunD Proto-Oncogene
LDA	Linear discriminant analysis
Lif	Leukemia inhibitory factor
MCP-1	Monocyte chemoattractant protein-1
MFM	Motor Function Measurement
MIP-1β	Macrophage inflammatory protein-1β
miRNAs	MicroRNAs
MMA/SDMA	Monomethylarginine/symmetric dimethylarginine
MOST	Multispectral optoacoustic tomography
MT	Muscle thickness
MUNE	Motor unit number estimation
MUNIX	Motor unit number index
myomiRs	Muscle-specific miRNAs
NCOR2	Nuclear receptor corepressor 2
NCX2	Sodium-calcium exchanger 2
NfH	Neurofilament heavy chain
NfL	Neurofilament light chain
NfM	Neurofilament medium chain
NFs	Neurofilaments
NF-κB	Nuclear factor kappa B
NPV	Negative predictive value
Pak4	P21-activated kinase 4
PCR	Polymerase chain reaction.
PLS3	Plastin 3
PME	Progressive myoclonic epilepsy
pNfH	Phosphorylated neurofilament heavy chain
PRMT	Protein arginine methyltransferase
Ptgs2	Prostaglandin-endoperoxide synthase 2
QAlb	Albumin quotient
RHOA	Ras homolog family member A
ROCK1	Rho-associated coiled-coil containing protein kinase 1
RULM	Revised upperlimb module
S100B	S100 calcium-binding protein B
sAPP	Soluble amyloid precursor protein
SD	Standard deviation
SIMOA	Sensitive single-molecule array
SLC23A2	Solute carrier family 23 member 2
SMA	Spinal muscular atrophy
SMA-PME	SMA with progressive myoclonic epilepsy
SMN	Survival motor neuron
snRNP	Small nuclear ribonucleoprotein
Syt	Synaptotagmin
SOX2	SRY-box transcription factor 2
TBXT	T-box transcription factor T
TNF-α	Tumor necrosis factor-alpha
TRADD	TNF Receptor-Associated Death Domain
WGCNA	Weighted correlation network analysis