Pharmaceutical preparations intended to treat rare diseases, namely, urea cycle disorders [, organic acidurias, spinal muscular atrophy, Huntington's disease, tuberculosis, amyotrophic lateral sclerosis, lymphoma, leukemia, maple syrup urine disease, thalassemia, sickle cell disease cystic fibrosis, alpha-1 antitrypsine deficiency ]

'-1':37 'aciduria':13 'alpha':36 'amyotroph':21 'antitrypsin':38 'atrophi':16 'cell':32 'cycl':10 'cystic':34 'defici':39 'diseas':7,19,29,33 'disord':11 'fibrosi':35 'huntington':17 'intend':3 'later':22 'leukemia':25 'lymphoma':24 'mapl':26 'muscular':15 'name':8 'organ':12 'pharmaceut':1 'prepar':2 'rare':6 'sclerosi':23 'sickl':31 'spinal':14 'syrup':27 'thalassemia':30 'treat':5 'tuberculosi':20 'urea':9 'urin':28

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