skysona
bluebird bio (netherlands) b.v. - elivaldogene autotemcel - adrenoleukodystrophy - other nervous system drugs - treatment of early cerebral adrenoleukodystrophy in patients less than 18 years of age, with an abcd1 genetic mutation, and for whom a human leukocyte antigen (hla) matched sibling haematopoietic stem cell donor is not available.
icatibant accord
accord healthcare s.l.u. - icatibant acetate - angioedēma, iedzimta - other hematological agents - icatibant accord is indicated for symptomatic treatment of acute attacks of hereditary angioedema (hae) in adults, adolescents and children aged 2 years and older, with c1 esterase inhibitor deficiency.
ozawade
bioprojet pharma - pitolisant - sleep apnea, obstructive - other nervous system drugs - ozawade is indicated to improve wakefulness and reduce excessive daytime sleepiness (eds) in adult patients with obstructive sleep apnoea (osa).
amifampridine serb
serb sa - amifampridine phosphate - lambert-eaton myasthenic syndrome; paraneoplastic syndromes, nervous system; nervous system neoplasms; paraneoplastic syndromes; nervous system diseases; autoimmune diseases of the nervous system; neurodegenerative diseases; neuromuscular diseases; neuromuscular junction diseases; immune system diseases; autoimmune diseases; autoimmune diseases of the nervous system; cancer; neoplasms - other nervous system drugs - simptomātiska lambert-eaton miaestētiskā sindroma (lems) ārstēšana pieaugušajiem.
amvuttra
alnylam netherlands b.v. - vutrisiran sodium - amyloid neuropathies, familial - other nervous system drugs - treatment of hereditary transthyretin-mediated amyloidosis (hattr amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy.
pyrukynd
agios netherlands b.v. - mitapivat sulfate - genetic diseases, inborn; anemia, hemolytic - other hematological agents - pyrukynd is indicated for the treatment of pyruvate kinase deficiency (pk deficiency) in adult patients (see section 4.
hemgenix
csl behring gmbh - etranacogene dezaparvovec - hemophilia b - other hematological agents - treatment of severe and moderately severe haemophilia b (congenital factor ix deficiency) in adult patients without a history of factor ix inhibitors.
sohonos
ipsen pharma - palovarotene - myositis ossificans - other drugs for disorders of the musculo-skeletal system - treatment of fibrodysplasia ossificans progressiva.
camzyos
bristol-myers squibb pharma eeig - mavacamten - cardiomyopathy, hypertrophic - other cardiac preparations - treatment of symptomatic obstructive hypertrophic cardiomyopathy.
gold collagen® forte 40+ (2019.) šķidrums
nikko yakuhin kogyo co., ltd. - šķidrums