Results 191 to 200 of about 88,273 (287)

Prevalence of low‐level viremia and its association with virological failure among adolescents living with HIV in Eastern Cape, South Africa

HIV Medicine, EarlyView.
Abstract Objectives Low‐level viremia (LLV) has been associated with an increased risk of virological failure among adults on antiretroviral therapy (ART). However, evidence on the clinical implications of LLV among adolescents living with HIV remains limited.
Zea Leon, Olanrewaju Edun, Siyanai Zhou, Janke Tolmay, Lucie Cluver, Gayle Sherman, Ahmad H. Mazanderani, Elona Toska   +7 more
wiley   +1 more source

Orphan Drugs, Compounded Medication and Pharmaceutical Commons. [PDF]

Front Pharmacol, 2021
Hendrickx K, Dooms M.
europepmc   +1 more source

An Australian standard of care for Niemann–Pick disease type C

Internal Medicine Journal, EarlyView.
Abstract Background Niemann–Pick disease type C (NP‐C) is the fifth most prevalent lysosomal disorder in Australia. Diagnostic delay is common, impacted by disease heterogeneity, limited awareness within clinical gateway services and exclusion from state‐based newborn screening programmes.
Michel Tchan, Nicholas Smith, Heidi Peters, Ellie Van Velsen, Catherine Marraffa, Carolyn Ellaway, Katrina Cruz, Shekeeb S. Mohammad, Maina Kava, Joy Yaplito‐Lee, Shanti Balasubramaniam, Yusof Rahman, Brendon Boot, Ashley Bush, Felicity Munro, Leniza Hamoy, Ya Hui Hung, Philippa Johnston, Deanna Carpino, Molly Williams, Sharmila Kiss, Rebecca Quin, Ingrid Sutherland, Mark Walterfang   +23 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

A Retrospective Chart Review of the Prevalence, Characteristics, and Discharge Destinations of Patients Hospitalized as “Social Admissions”

Journal of the American Geriatrics Society, EarlyView.
Overview of study.
Jasmine Mah, Lucy Eum, Lindsey Puddicombe, Christie Stilwell, Karen Nicholls, Hamish Frayne, Clara Long, Kristin Ko, Victoria Howatt, Margaret Man‐Ger Sun, Lynnea Lobert, Gali Latariya, Katalin Koller, Susan Freter, Maia von Maltzahn, Kenneth Rockwood, Samuel D. Searle, Melissa K. Andrew   +17 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

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

Narcolepsy and rapid eye movement sleep

Journal of Sleep Research, Volume 34, Issue 2, April 2025.
Summary Since the first description of narcolepsy at the end of the 19th Century, great progress has been made. The disease is nowadays distinguished as narcolepsy type 1 and type 2. In the 1960s, the discovery of rapid eye movement sleep at sleep onset led to improved understanding of core sleep‐related disease symptoms of the disease (excessive ...
Francesco Biscarini, Lucie Barateau, Fabio Pizza, Giuseppe Plazzi, Yves Dauvilliers   +4 more
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

Crowd Funding for Orphan Drugs: The Case of Baby Pia. [PDF]

Front Pharmacol, 2021
Pinxten W.
europepmc   +1 more source