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patients with duchenne muscular dystrophy lack normal dystrophin, a protein found in muscles.
pazjenti b’distrofija muskolari duchenne għandhom nuqqas ta’ distrofin normali, proteina li tinstab fil- muskoli.
care nmd: dissemination and implementation of the standards of care for duchene muscular dystrophy in europe
care nmd: it-tixrid u l-implimentazzjoni tal-istandards tal-kura għad-distrofija muskolari duchene fl-ewropa
duchenne muscular dystrophy is a genetic disease that gradually causes weakness and loss of muscle function.
distrofija muskolari duchenne hija marda ġenetika li gradwalment tikkawża dgħjufija u telf tal-funzjoni tal-muskoli.
the committee also acknowledged the seriousness of duchenne muscular dystrophy and the unmet medical need of patients with this condition.
il-kumitat irrikonoxxa wkoll il- gravità tad-distrofija muskulari duċhenne u l-ħtieġa medika mhux milħuqa ta’ pazjenti b’din il- kundizzjoni.
translarna is used to treat duchenne muscular dystrophy resulting from a specific genetic defect that affects normal muscle function.
translarna jintuża biex jikkura distrofija muskolari ta’ duchenne li tirriżulta minn difett ġenetiku speċifiku li jaffettwa l-funzjoni normali tal-muskoli.
treatment with translarna should only be initiated by specialist physicians with experience in the management of duchenne/becker muscular dystrophy.
il-kura b’translarna għandha tinbeda biss minn tobba b’esperjenza fil-ġestjoni ta’ distrofija muskulari ta’ duchenne/becker.
duchenne muscular dystrophy is caused by genetic changes that result in an abnormality in a muscle protein called dystrophin which is needed for muscles to work properly.
id-distrofija muskolari ta’ duchenne hija kkawżata minn bidliet ġenetiċi li jirriżultaw f’anormalità fi proteina tal-muskolu msejħa dystrophin li hija meħtieġa għall-muskoli biex jaħdmu sew.
translarna can only be obtained with a prescription and treatment should be started by a specialist doctor experienced in the management of duchenne/becker muscular dystrophy.
translarna jista’ jinkiseb biss b’riċetta ta’ tabib u l-kura għandha tinbeda minn tabib speċjalista b’esperjenza fil-ġestjoni ta’ distrofija muskolari duchenne/becker.
because this protein helps to protect muscles from injury as muscles contract and relax, in patients with duchenne muscular dystrophy the muscles become damaged and eventually stop working.
minħabba li din il-proteina tgħin tipproteġi l-muskoli minn ħsara waqt li l-muskoli jikkontrattaw u jirrilassaw, f’pazjenti b’distrofija muskolari duchenne, il-muskoli ssirilhom il-ħsara u eventwalment jieqfu jaħdmu.
translarna is indicated for the treatment of duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 5 years and older (see section 5.1).
translarna huwa indikat għall-kura ta’ distrofija muskolari ta’ duchenne li tirriżulta minn mutazzjoni nonsense fil-ġene ta’ dystrophin, f’pazjenti ambulatorji li għandhom 5 snin u aktar (ara sezzjoni 5.1).
careful monitoring of patients with compromised corneas, such as patients with diabetes mellitus or corneal dystrophies, is recommended.
hija rrakkomandata sorveljanza b’attenzjoni ta’ pazjenti b’korneji kompromessi, bħal pazjenti b’dijabete mellitus jew distrofiji tal-korneji.