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. 2020 Oct 30;8:593282. doi: 10.3389/fped.2020.593282

Table 1.

Risk levels and susceptible age groups of acute respiratory compromises in different neuromuscular disorders.

Primarily affected age group Risk level of ARF occurrence Affected NMD
At birth or within the first year of life Usually inevitable if untreated Spinal muscular atrophy (SMA) type 1 (if untreated)*
Spinal muscular atrophy with respiratory distress (SMARD)
Congenital myotonic dystrophy (type 1)
Infantile Pompe disease (if untreated)*
Some congenital myopathies (e.g., neonatal form of nemaline myopathy, minicore myopathy, and X-linked myotubular myopathy)
Some congenital muscular dystrophies (CMD) (e.g., Walker-Warburg syndrome and Muscle–eye–brain disease)
Some mitochondrial diseases
Some congenital myasthenic syndromes
Infant-to-adult life Very high risk Some limb-girdle muscular dystrophy (LGMD), especially with sarcoglycanopathies (LGMD types 2C, 2D, 2E, 2F) and LGMD type 2I
Some CMD, especially merosin negative types 1A, 1B, 1C
Some myofibrillar myopathies (e.g., hereditary myopathy with early respiratory failure)
Early-onset infantile facioscapulohumeral muscular dystrophy (FSHD)
Early-onset Charcote-Marie-Tooth disease (CMTD) especially with GDAP1 mutation
Some congenital myopathies (e.g., severe recessive type of central core myopathy)
infant-to-adult life High risk Duchenne muscular dystrophy (DMD), usually after second decade
SMA type 2
Myotonic dystrophy type 1 (DM1)
Late-onset Pompe disease (LOPD)
Some CMD (e.g., Ullrich type, and Fukuyama congenital muscular dystrophy)
Some LGMD (e.g., calpainopathy)
Some congenital myopathies (e.g., centronuclear myopathy)
Bethlem myopathy
Congenital myasthenic syndromes
Some mitochondrial myopathies (e.g., A3243G mutation in the tRNALeu gene)
Intermediate risk Becker muscular dystrophy (BMD)
SMA type 3
Inflammatory myopathies (e.g., polymyositis, dermatomyositis)
Classical type of FSHD
Some types of Charcot–Marie–Tooth disease (e.g., CMTD type 1B and 4)
Some congenital myopathies
Some mitochondrial myopathies
Guillain–Barré syndrome (GBS)
Myasthenia gravis (MG)
Low risk Oculopharyngeal muscular dystrophy (OPMD)
Other types of CMTD
Chronic inflammatory demyelinating polyneuropathy (CIDP)
*

Novel therapies are currently available (e.g., enzyme replacement, antisense nucleotide, and gene therapy) to be delivered in the neonatal period.

Data of this table are modified and summarized from references: (1113).