Results 111 to 120 of about 22,997 (249)

Access to orphan drugs in adults with inherited metabolic diseases in Switzerland: a single-center retrospective cohort study. [PDF]

Orphanet J Rare Dis
Antoni G, Kumps C, Berger J, Barman-Aksözen J, Tran C.   +4 more
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

Optimizing regional rare disease management systems within China's policy framework: practical insights from the shantou experience. [PDF]

Orphanet J Rare Dis
Wu B, Wang Y, Liu C, Wang Q, Gong M, Chen Y.   +5 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

A cost-based-plus pricing approach for repurposed tiratricol in the treatment of Allan-Herndon-Dudley syndrome. [PDF]

Orphanet J Rare Dis
Manders E, Keestra S, Bannenberg W, van der Wel V, de Visser S, Hollak C.   +5 more
europepmc   +1 more source

Milestones On Orphan Medicinal Products Development: The 100 First Drugs for Rare Diseases Approved Throughout Europe [PDF]

Clinical Therapeutics, 2015
C. Pontes, J.M. Fontanet, M. Gomez-Valent, J. Rios Guillermo, R. Vives Vilagut, R. Morros, J. Martinalbo, J. Torrent-Farnell, F Torres   +8 more
openaire   +2 more sources

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

Use of Real-World Evidence in Health Technology Assessments of Non-Oncology Rare Disease Therapies. [PDF]

J Mark Access Health Policy
Blandy O, Lembo P, Folorunso R, Myren KJ, Chevrou-Severac H, Thomas SK.   +5 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

Orphan medical devices: addressing the regulatory and access gaps in the EU and US. [PDF]

Front Public Health
Akodad S, Haon B, Rochaix L, Stevens H.
europepmc   +1 more source