Australija - engleski - Department of Health (Therapeutic Goods Administration)

recordati rare diseases australia pty ltd - pasireotide embonate, quantity: 82.26 mg (equivalent: pasireotide, qty 60 mg) - injection, modified release - excipient ingredients: polyglactin glucose; polyglactin - signifor lar is indicated for the treatment of adult patients with acromegaly for whom surgery is not an option or has not been curative or who are inadequately controlled on treatment with other somatostatin analogues.

Australija - engleski - Department of Health (Therapeutic Goods Administration)

recordati rare diseases australia pty ltd - pasireotide embonate, quantity: 54.84 mg (equivalent: pasireotide, qty 40 mg) - injection, modified release - excipient ingredients: polyglactin glucose; polyglactin - signifor lar is indicated for the treatment of adult patients with acromegaly for whom surgery is not an option or has not been curative or who are inadequately controlled on treatment with other somatostatin analogues.

Australija - engleski - Department of Health (Therapeutic Goods Administration)

recordati rare diseases australia pty ltd - pasireotide embonate, quantity: 27.42 mg (equivalent: pasireotide, qty 20 mg) - injection, modified release - excipient ingredients: polyglactin glucose; polyglactin - signifor lar is indicated for the treatment of adult patients with acromegaly for whom surgery is not an option or has not been curative or who are inadequately controlled on treatment with other somatostatin analogues.

Malta - engleski - Medicines Authority

recordati rare disease immeuble “le wilson”, 70, avenue du général de gaulle, f-92800 puteaux, france - human hemin - concentrate for solution for infusion - human hemin 25 mg/ml - other hematological agents

Australija - engleski - Department of Health (Therapeutic Goods Administration)

recordati rare diseases australia pty ltd - ibuprofen, quantity: 5 mg/ml - injection, solution - excipient ingredients: trometamol; sodium hydroxide; sodium chloride; hydrochloric acid; water for injections - pedea is indicated for the treatment of haemodynamically significant patent ductus arteriosus in preterm newborn infants less than 34 weeks of gestational age.

Australija - engleski - Department of Health (Therapeutic Goods Administration)

recordati rare diseases australia pty ltd - dactinomycin, quantity: 500 microgram - injection, powder for - excipient ingredients: mannitol - wilm's tumour; rhabdomyosarcoma; carcinoma of the testes and uterus. other neoplasms: actinomycin d given iv or by regional perfusion, either alone or with other antineoplastic compounds or with x-ray therapy in the palliative treatment of ewing's sarcoma and sarcoma botryoides; non-metastatic ewing's carcinoma. actinomyin d and radiation therapy; actinomycin d in various types of sarcoma, carcinoma and adenocarcinoma using isolation-perfusion technique. for full list of indications refer to approved pi document.

Sjedinjene Američke Države - engleski - NLM (National Library of Medicine)

recordati rare diseases - carglumic acid (unii: 5l0hb4v1ew) (carglumic acid - unii:5l0hb4v1ew) - carbaglu is indicated in adult and pediatric patients as: - adjunctive therapy to standard of care for the treatment of acute hyperammonemia due to nags deficiency. - maintenance therapy for the treatment of chronic hyperammonemia due to nags deficiency. carbaglu is indicated in adult and pediatric patients as adjunctive therapy to standard of care for the treatment of acute hyperammonemia due to pa or mma. none pregnancy exposure registry there is a pregnancy exposure registry that monitors pregnancy outcomes in women with nags deficiency exposed to carbaglu.  if carbaglu is administered during pregnancy, health care providers should report carbaglu exposure by calling 1-888-575-8344. risk summary although rare case reports of carbaglu use in pregnant women are insufficient to inform a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes, untreated nags deficiency, pa and mma can result in irreversible neurologic damage and death in pregnant women (see clinical considerations). in an animal reproduction study, decreased survival and growth occurred in offspring born to rats that received carglumic acid at a dose approximately 38 times the maximum reported human maintenance dose. the estimated background risk of major birth defects and miscarriage for the indicated population is unknown. all pregnancies have a background risk of birth defect, miscarriage, or other adverse outcomes. in the u.s. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively. clinical considerations disease-associated maternal and/or embryo/fetal risk pregnant women with urea cycle disorders, pa, and mma may experience an increase in catabolic stress which can trigger a hyperammonemic crisis both in the intrapartum and in the post-partum (3-14 days post-partum) periods. maternal complications related to hyperammonemic crisis can include neurological impairment, coma and in some cases death.  data animal data no effects on embryo-fetal development were observed in pregnant rats treated with up to 2000 mg/kg/day (approximately 38 times the maximum reported human maintenance dose [100 mg/kg/day] based on auc [area under the plasma concentration-time curve]) from two weeks prior to mating through organogenesis or in pregnant rabbits treated with up to 1000 mg/kg/day (approximately 6 times the maximum reported human maintenance dose [100 mg/kg/day] based on auc) during organogenesis. in a pre- and post-natal developmental study, female rats received oral carglumic acid from organogenesis through lactation at doses of 500 mg/kg/day and 2000 mg/kg/day. decreased growth of offspring was observed at 500 mg/kg/day and higher (approximately 38 times the maximum reported human maintenance dose [100 mg/kg/day] based on auc), and reduction in offspring survival during lactation was observed at 2000 mg/kg/day (approximately 38 times the maximum reported human maintenance dose [100 mg/kg/day] based on auc). no effects on physical and sexual development, learning and memory, or reproductive performance were observed through maturation of the surviving offspring at maternal doses up to 2000 mg/kg/day. the high dose (2000 mg/kg/day) produced maternal toxicity (impaired weight gain and approximately 10% mortality). risk summary it is not known whether carglumic acid is present in human milk. there are no available data on the effects of carglumic acid on the breastfed infant or the effects on milk production. carglumic acid is present in milk from treated rats. when a drug is present in animal milk, it is likely that the drug will be present in human milk. the developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for carbaglu and any potential adverse effects on the breastfed child from carbaglu or from the underlying maternal condition. the safety and effectiveness of carbaglu for the treatment of pediatric patients (birth to 17 years of age) with acute or chronic hyperammonemia due to nags deficiency and acute hyperammonemia due to pa or mma have been established, and the information on these uses are discussed throughout the labeling. there are insufficient data to determine if there is a difference in clinical or biochemical responses between adult and pediatric patients treated with carbaglu. clinical studies of carbaglu did not include patients 65 years of age and older to determine whether they respond differently from younger patients. plasma concentrations of carglumic acid increased in patients with renal impairment [see clinical pharmacology (12.3)] . reduce the carbaglu dosage in patients with moderate or severe renal impairment [see dosage and administration (2.4)]. the pharmacokinetics of carglumic acid have not been evaluated in patients with end stage renal disease.

Sjedinjene Američke Države - engleski - NLM (National Library of Medicine)

recordati rare diseases, inc. - mechlorethamine hydrochloride (unii: l0mr697hhi) (mechlorethamine - unii:50d9xsg0vr) - mechlorethamine hydrochloride 10 mg in 10 ml

Sjedinjene Američke Države - engleski - NLM (National Library of Medicine)

recordati rare diseases, inc. - cysteamine hydrochloride (unii: if1b771svb) (cysteamine - unii:5ux2sd1ke2) - cystadrops is a cystine-depleting agent indicated for the treatment of corneal cystine crystal deposits in adults and children with cystinosis. none. risk summary there are no adequate and well-controlled studies of ophthalmic cysteamine in pregnant women to inform any drug associated risks. oral administration of cysteamine to pregnant rats throughout the period of organogenesis was teratogenic at doses 240 to 960 times the recommended human ophthalmic dose (based on body surface area) [ see data] . cystadrops should be used during pregnancy only if the potential benefit justifies the potential risk to the fetus. the estimated background risk of major birth defects and miscarriage for the indicated population is unknown. all pregnancies have a background risk of birth defect, loss, or other adverse outcomes in the u.s. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively. data animal data teratology studies have been performed in rats at oral doses in the range of 37.5 mg/kg/day to 150 mg/kg/day (240 to 960 times the recommended human ophthalmic dose based on body surface area) and have shown cysteamine bitartrate to be teratogenic. observed teratogenic findings were intrauterine death, cleft palate, kyphosis, heart ventricular septal defects, microcephaly, exencephaly, and growth deficits. risk summary there is no information regarding the presence of cysteamine in human milk, the effects on the breastfed infants, or the effects on milk production. cysteamine administered orally is present in milk of lactating rats. it is not known whether measurable levels of cysteamine would be present in maternal milk following topical ocular administration of cystadrops. the developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for cystadrops and any potential adverse effects on the breastfed child from cystadrops or from the underlying maternal conditions. the safety and effectiveness of cystadrops has been established in pediatric patients. use of cystadrops is supported by adequate and well controlled trials in pediatric patients and additional experience supporting the safety of cystadrops. clinical studies of cystadrops did not include sufficient numbers of subjects aged 65 and over to determine whether they respond differently from younger subjects. other reported clinical experience has not identified differences in responses between the elderly and younger patients.  the effect of renal impairment on the pharmacokinetics of cysteamine following ophthalmic administration of cysteamine ophthalmic solution has not been evaluated. clearance of cysteamine from the conjunctival sac of the eye is not dependent on renal function and the total systemic dose is negligible, so impaired renal function is unlikely to affect total body clearance. the total daily ophthalmic dose is less than 4% of the recommended oral daily dose of cysteamine; thus, the systemic exposure following ophthalmic administration is expected to be negligible compared to oral administration.

Sjedinjene Američke Države - engleski - NLM (National Library of Medicine)

recordati rare diseases - betaine (unii: 3scv180c9w) (betaine - unii:3scv180c9w) - betaine 1 g in 1 g - cystadane ® is indicated for the treatment of homocystinuria to decrease elevated homocysteine blood concentrations in pediatric and adult patients. included within the category of homocystinuria are: - cystathionine beta-synthase (cbs) deficiency - 5,10-methylenetetrahydrofolate reductase (mthfr) deficiency - cobalamin cofactor metabolism (cbl) defect none. risk summary available data from a limited number of published case reports and postmarketing experience with cystadane use in pregnancy have not identified any drug associated risks for major birth defects, miscarriage, or adverse maternal or fetal outcomes. animal reproduction studies have not been conducted with betaine. the estimated background risk of major birth defects and miscarriage for the indicated population is unknown. all pregnancies have a background risk of birth defect, loss, or other adverse outcomes. in the u.s. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnanc