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Bicuspid aortic valve ( BAV ) is the most frequent congenital heart disease with frequent involvement in thoracic aortic dilatation, aneurysm and dissection. Although BAV and Marfan syndrome ( MFS ) s ...


The FDA ( Food and Drug Administration ) has approved Sylvant ( Siltuximab ) for the treatment of patients with multicentric Castleman’s disease ( MCD ) who are human immunodeficiency virus ( HIV ) ne ...


The application for marketing authorisation of Nintedanib, a tyrosine kinase inhibitor ( TKI ), for the treatment of idiopathic pulmonary fibrosis ( IPF ) has been validated and granted accelerated as ...


The involvement of optic and auditory pathways has rarely been studied in demyelinating polyneuropathies. A cohort of patients with acquired and genetic demyelinating neuropathy has been studied. T ...


The aim of a study was to determine whether the clinical characteristics and electrodiagnostic classification of nerve injury, and response to treatment differed in patients diagnosed with chronic inf ...


Lymphangioleiomyomatosis ( LAM ), sporadic or in women with tuberous sclerosis complex ( TSC ), is characterized by cystic lung destruction, lymphatic involvement ( eg, chylous pleural effusions, lymp ...


Sarepta Therapeutics has announced new pulmonary function data through week 120 from Study 202, a Phase IIb open-label extension study of eteplirsen in patients with Duchenne muscular dystrophy ( DMD ...


Sarepta Therapeutics has announced data through week 120 from Study 202, a phase IIb open-label extension study of Eteplirsen in patients with Duchenne muscular dystrophy ( DMD ). Results through more ...


The FDA ( Food and Drug Administration ) has approved Myalept ( Metreleptin for injection ) as replacement therapy to treat the complications of leptin deficiency, in addition to diet, in patients wit ...


Although various modalities of treatment of chronic inflammatory demyelinating polyradiculopathy ( CIDP ) there are not any treatment protocol agreed. Researchers have retrospectively evaluated the ...


Office of Orphan Products Development ( OOPD ) of the Food and Drug Administration ( FDA ) has granted orphan drug designation for drug candidate, Erdosteine, for the treatment of bronchiectasis. Erdo ...


The FDA ( Food and Drug Administration ) has granted an orphan drug designation ( ODD ) to Soliris ( Eculizumab ), a first-in-class terminal complement inhibitor, for the prevention of delayed graft f ...


BioMarin has announced that the FDA ( Food and Drug Administration ) has approved Vimizim ( Elosulfase alfa ) for patients with mucopolysaccharidosis type IVA ( MPS IVA; Morquio A syndrome ).Vimizim i ...


Pallister-Killian syndrome ( PKS ) is a sporadic multisystem genetic diagnosis characterized by facial dysmorphia, variable developmental delay and intellectual impairment, hypotonia, hearing loss, se ...


Chronic inflammatory demyelinating polyradiculoneuropathy ( CIDP ) causes progressive or relapsing weakness and numbness of the limbs, developing over at least two months. Uncontrolled studies sugge ...