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Table 1 Sampled rare diseases and medicines

From: Catastrophic expenditure and impoverishment of patients affected by 7 rare diseases in China

Rare disease

Prevalence

Therapeutic regimen

Health expenditure per year (1,000 yuan)

Health expenditures as a share of per capita GDP

Duchenne Muscular Dystrophy (DMD)

5/100000

Methylprednisolone Tablet, 0.6 mg/Kg/day

1.20

0–1

Alport Syndrome (AS)

2/100000

ACEI combined with ARB

7.50

0–1

Tuberous Sclerosis Complex (TSC)

8.8/100000

Sirolimus Tablet, 1 mg bid

40

0–1

Amyotrophic Lateral Sclerosis (ALS)

5/100000

Riluzole Tablet, 50 mg bid

55

1–3

Idiopathic Pulmonary Fibrosis (IPF)

16.7/100000

Pirfenidone Tablet, 200–400 mg tid

86

1–3

Pulmonary Arterial Hypertension (PAH)

5/100000

Bosentan Tablet, 125 mg bid

240

>3

Gaucher Disease (GD)

1/100000

Imiglucerase for Injection, 60 U/kg, fortnightly

2000

>3