Results 151 to 160 of about 1,419,402 (298)

Real‐World Safety and Effectiveness of Icatibant in Patients With Hereditary Angioedema (HAE): Post‐Marketing Surveillance in Japan

The Journal of Dermatology, EarlyView.
ABSTRACT Hereditary angioedema (HAE) is a rare, autosomal dominant, life‐threatening disease characterized by recurrent attacks of swelling of the face, upper airway, gastrointestinal tract, limbs, and/or genitalia. Genetic mutations of the SERPING1 gene encoding the C1‐inhibitor (C1‐INH) plasma protein are associated with HAE‐C1INH.
Kumiko Hosono, Miyako Hoshino
wiley   +1 more source

Stretching Scarce Authorizing Legislation as Far as Possible: A Legislative History of the 340B Drug Pricing Program

The Milbank Quarterly, EarlyView.
Policy Points The original purpose of the 340B program was to exempt Public Health Service Act funded clinics and state and local public hospitals from the inflationary best‐price component of the recently enacted Medicaid drug rebate program. The secondary purpose was to reduce drug prices for these clinics and hospitals in order to preserve and ...
SAYEH NIKPAY, MIKAYLA REINKE, NICOLE QUINONES   +2 more
wiley   +1 more source

How to START? Four pillars to optimally begin your orphan drug development. [PDF]

Orphanet J Rare Dis, 2023
Jonker AH, Batista L, Gabaldo M, Hivert V, Ardigo D, IRDiRC ODDG TF and IRDiRC TSC.   +5 more
europepmc   +1 more source

Tick‐Borne Encephalitis (TBE) Vaccine in the National Immunisation Programme—For Whom, When and Where?

Acta Paediatrica, EarlyView.
ABSTRACT The incidence of Tick‐borne encephalitis (TBE) cases has increased. The presumed location of transmission of Tick‐borne encephalitis virus (TBEV) has been expanding increasingly in the western parts of Europe during the past decade. There has also been an increased incidence of surveillance‐reported TBE cases in southern Sweden and southern ...
H. H. Askling, D. Zavadska
wiley   +1 more source

Size Does Matter! Prioritizing Rare Diseases for Luck Egalitarian Reasons

Bioethics, EarlyView.
ABSTRACT Rarity provides a challenging case for contemporary priority setting. On the one hand, many philosophers and economists argue that rarity has no inherent moral value, and thus that rare diseases merit no special treatment in priority setting decisions simply because they are rare.
Didde Boisen Andersen, Andreas Brøgger Albertsen, Sarah Wadmann, Mickael Bech   +3 more
wiley   +1 more source

Avatrombopag for persistent and chronic immune thrombocytopenia in children: A post hoc analysis of clinical characteristics of response from a phase 3b clinical trial

British Journal of Haematology, EarlyView.
Summary Avatrombopag treatment resulted in significantly higher platelet response rates in patients when compared with placebo in a phase 3b study of patients with paediatric immune thrombocytopenia (ITP) (NCT04516967). These post hoc analyses of the 12‐week core phase further assessed the efficacy of avatrombopag, considering the influence of baseline
Rachael F. Grace, Göksel Leblebisatan, Yesim Aydinok, Sule Unal, John Grainger, Amanda B. Grimes, Michele P. Lambert, Jessica Zhang, Brian D. Jamieson, Faizan Sattar, Scott Kolodny, Michael Vredenburg, on behalf of the AVA‐PED‐301 Study Group   +12 more
wiley   +1 more source

Advancing hypoparathyroidism treatment: FDA approval of Palopegteriparatide as a promising orphan drug. [PDF]

Ann Med Surg (Lond)
Shahid SR, Shahid K, Ehsan S, Khalid M, Waafira A.   +4 more
europepmc   +1 more source

Orphan drugs' clinical uncertainty and prices: Addressing allocative and technical inefficiencies in orphan drug reimbursement. [PDF]

Front Pharmacol, 2023
Eichler HG, Kossmeier M, Zeitlinger M, Schwarzer-Daum B.   +3 more
europepmc   +1 more source

Is there a role for cannabidiol in obesity, metabolic syndrome and binge eating?

British Journal of Pharmacology, EarlyView.
Cannabidiol (CBD) is one of the most abundant phytocannabinoids isolated from the Cannabis sativa plant. CBD is a lipophilic, non‐intoxicating substance that differently from Δ9‐tetrahydrocannabinol (Δ9‐THC) does not present the typical profile of a drug of abuse.
Luca Botticelli, Emanuela Micioni Di Bonaventura, Giacomo Einaudi, Gustavo Provensi, Alessia Costa, Claudio D'Addario, Carlo Cifani, Maria Vittoria Micioni Di Bonaventura   +7 more
wiley   +1 more source

W., Joe (Box 91, Folder 20) [PDF]

Series VIII: Audiocassettes features informal oral histories about the Hebrew Orphan Asylum and its alumni.Digital recordingDigital finding ...
Hebrew Orphan Asylum of New York
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