Results 151 to 160 of about 32,298 (248)

Financing of Rare Diseases and Orphan Drugs in A Sanctioned Country: A Qualitative Study. [PDF]

Med J Islam Repub Iran, 2022
Naghdi S, Maleki M, Vatankhah S.
europepmc   +1 more source

Estimating the minimal cost of delivering nutrition‐specific and nutrition‐sensitive interventions in Ethiopia

Maternal &Child Nutrition, EarlyView.
The minimum cost of the 10 years on identified nutrition‐specific and nutrition‐sensitive interventions of the National Food and Nutrition Strategy in Ethiopia is estimated to be US$ 2.55bn with an average annual cost of $250 million over 10 years (2021–2030), which is only 2.3% of the Ethiopian Annual GDP 111.27 billion US dollars in 2021 (World bank).
Yetayesh Maru, Firehiwot Mesfin, Arnaud Laillou, Ramadhani Noor, Shiva Raj Adhikari, Meseret Zelalem, Hiwot Darsene, Solomon Memire, Andarge Abie, Stanley Chitekwe   +9 more
wiley   +1 more source

Controlled access to lumasiran in primary hyperoxaluria type 1: evaluation of a new access route for orphan drugs in the Netherlands. [PDF]

Nephrol Dial Transplant
Deesker LJ, Franssen CFM, Dorresteijn E, van de Kar NCAJ, Nurmohamed SA, Severs D, Garrelfs SF, Pisters-van Roy AAMG, Hollak CEM, Groothoff JW.   +9 more
europepmc   +1 more source

Assessing the value of orphan drugs using conventional cost-effectiveness analysis: Is it fit for purpose? [PDF]

Orphanet J Rare Dis, 2022
Postma MJ, Noone D, Rozenbaum MH, Carter JA, Botteman MF, Fenwick E, Garrison LP.   +6 more
europepmc   +1 more source

TMEM16A chloride channels in the female reproductive tract and their role in normal and dysfunctional pregnancy and labour

The Journal of Physiology, EarlyView.
Abstract figure legend TMEM16A plays a pivotal role in setting the duration of the action potential plateau in human uterine smooth muscle. An increase in TMEM16A expression in labour underpins a lengthening of the plateau and this provides time for the contraction to become larger and longer, important for timely successful labour.
Helena C. Parkington, Harold A. Coleman, Penelope M. Sheehan, Shaun P. Brennecke, Marianne Tare   +4 more
wiley   +1 more source

Recommended methods for the collection of clinical expert judgment in rare diseases: Generating evidence to support reimbursement of orphan drugs. [PDF]

Int J Technol Assess Health Care
Griffiths A, Dunning L, Facey K, Jankovic D, González Malla C, Low E, Meregaglia M, Schmidt F, Wilson K, Upadhyaya S, Chapman N, Zentai C, Wright S, Newell I.   +13 more
europepmc   +1 more source

Cannabinoid exposure during pregnancy: Cardiorespiratory effects and offspring outcomes

The Journal of Physiology, EarlyView.
Abstract figure legend Prenatal exposure to cannabinoids has been investigated across human and animal studies to understand its impact on physiological development. Evidence suggests that early‐life cannabinoid exposure influence multiple developmental processes, extending beyond neurodevelopmental outcomes to potentially affect placental function ...
Luis Gustavo A. Patrone, Luciane H. Gargaglioni   +1 more
wiley   +1 more source

Expediting treatments in the 21st century: orphan drugs and accelerated approvals. [PDF]

Orphanet J Rare Dis
Domike R, Raju GK, Sullivan J, Kennedy A.   +3 more
europepmc   +1 more source

Hypoxia and the cytoskeleton

The Journal of Physiology, EarlyView.
Abstract figure legend Schematic outlining the activation of hypoxia‐sensitive pathways, the influence of hypoxia and associated pathways on the cytoskeleton, and the impact of these on disease progression. Abstract A highly‐regulated and dynamic cytoskeleton is vital for functional cellular physiology and the maintenance of homeostasis.
Darragh Flood, Cormac T. Taylor
wiley   +1 more source

Determinants of the Financial Impact of Orphan Drugs in Italy: Differences Between Expected and Observed Pharmaceutical Expenditure. [PDF]

Drugs Real World Outcomes
Mennini FS, Cicchetti A, Sciattella P, Rumi F, Zanuzzi M, Carletto A, Sammarco A, Romano F, Russo P.   +8 more
europepmc   +1 more source