Eiropas Savienība - latviešu - EMA (European Medicines Agency)

orchard therapeutics (netherlands) bv - atidarsagene autotemcel - leukodystrophy, metachromatic - other nervous system drugs - libmeldy is indicated for the treatment of metachromatic leukodystrophy (mld) characterized by biallelic mutations in the arysulfatase a (arsa) gene leading to a reduction of the arsa enzymatic activity:in children with late infantile or early juvenile forms, without clinical manifestations of the disease,in children with the early juvenile form, with early clinical manifestations of the disease, who still  have the ability to walk independently and before the onset of cognitive decline.

Eiropas Savienība - latviešu - EMA (European Medicines Agency)

biocryst ireland limited - berotralstat dihydrochloride - angioedēma, iedzimta - other hematological agents - orladeyo is indicated for routine prevention of recurrent attacks of hereditary angioedema (hae) in adult and adolescent patients aged 12 years and older.

Eiropas Savienība - latviešu - EMA (European Medicines Agency)

roche registration gmbh  - risdiplam - muskuļu atrofija, mugurkaula - other drugs for disorders of the musculo-skeletal system - evrysdi is indicated for the treatment of 5q spinal muscular atrophy (sma) in patients with a clinical diagnosis of sma type 1, type 2 or type 3 or with one to four smn2 copies.

Eiropas Savienība - latviešu - EMA (European Medicines Agency)

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.

Eiropas Savienība - latviešu - EMA (European Medicines Agency)

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.

Eiropas Savienība - latviešu - EMA (European Medicines Agency)

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).

Eiropas Savienība - latviešu - EMA (European Medicines Agency)

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.

Eiropas Savienība - latviešu - EMA (European Medicines Agency)

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.

Eiropas Savienība - latviešu - EMA (European Medicines Agency)

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.

Eiropas Savienība - latviešu - EMA (European Medicines Agency)

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.