Teixeira de Barros, Carla Maria published the artcileTherapeutic profile of orphan medicines, Category: catalysis-chemistry, the publication is Pharmacoepidemiology and Drug Safety (2007), 16(4), 435-440, database is CAplus and MEDLINE.
Purpose: To characterize the therapeutic profile of orphan medicines. Materials and Methods: A cross-sectional study was performed during 2 mo in a convenience sample of seven hospital pharmacy services, in the region of Lisbon. Data were collected, from pharmaceutical service’s records. Results: A total of 18 orphan medicines, were dispensed to 355 patients with rare diseases. Most patients were adults (76.4%). Premature and neonates accounted with 50.0% of the paediatric patients. Differences were not found between the proportion of male and female patients across age groups (p = 0.762). Only 18.3% were inpatients. A high proportion of paediatric inpatients (58.3%) were seen in relation to adult inpatients (5.9%) (p = 0.001). In general, anti-neoplastic and immunomodulating agents for rare cancers were the most frequent dispensed medicines (51.3%). In relation to paediatrics, Caffeine Citrate for primary apnoea of premature newborns had the higher frequency distribution (57.1%). Five (71.4%) medicines dispensed for paediatrics, do not have market authorisation and the remaining (28.6%) were used off-label. For pulmonary arterial hypertension 19 of 27 patients (70.3%) were treated with Bosentan. According to evidence-based clin. practice guidelines, Bosentan has a good level of evidence and substantial benefit: grade of recommendation A. Conclusions: Most orphan medicines dispensed to paediatrics and adults were not licensed. A special pharmacovigilance program and a risk management plan through the entire life cycle should be implemented, towards effectiveness and safety of orphan medicines.
Pharmacoepidemiology and Drug Safety published new progress about 38260-01-4. 38260-01-4 belongs to catalysis-chemistry, auxiliary class Chelating Agents, name is N1,N1′-(Ethane-1,2-diyl)bis(ethane-1,2-diamine) dihydrochloride, and the molecular formula is C19H21N3O3S, Category: catalysis-chemistry.
Referemce:
https://courses.lumenlearning.com/boundless-chemistry/chapter/catalysis/,
Catalysis – Wikipedia