Results 191 to 200 of about 88,754 (289)

Hypoxia regulates Th17/Treg balance by altering chromatin accessibility and gene expression

The FEBS Journal, EarlyView.
T‐cell differentiation into Th17 or iTreg cells is key to inflammation control. Integrated RNA‐seq and ATAC‐seq analyses reveal that hypoxia induces more extensive gene expression changes in iTreg cells. Elevated levels of Hif1a and Stat3, along with increased accessibility to key transcription factor motifs, may predispose these cells toward ...
Mariana Cázares‐Olivera, Shiyang Song, Sofia Ylinen, Talha Qureshi, Bin Li, Zhi Chen   +5 more
wiley   +1 more source

HAEMFIX: Impact of Switching From SHL‐FIX to EHL‐FIX in Patients With Haemophilia B

Haemophilia, EarlyView.
ABSTRACT Introduction Haemophilia B is an X‐linked recessive bleeding disorder caused by coagulation factor IX (FIX) deficiency. Treatment involves intravenous replacement of FIX. Recently, extended half‐life (EHL) FIX products have been introduced alongside standard half‐life (SHL) products to optimize therapy. Aim This study evaluated bleeding rates,
Jasmin Lonardi, Susan Halimeh, Sylvia von Mackensen, Lisa Kleinlein, Juliet Fleischer, Henri Funk, Julia Hölz, Johannes Holzapfel, Sabrina Juranek, Victoria Lieftüchter, Christoph Bidlingmaier, Martin Olivieri   +11 more
wiley   +1 more source

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

Front Pharmacol, 2021
Hendrickx K, Dooms M.
europepmc   +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

In Silico Assessment of Limited Blood Sampling Strategies for Individualised Pharmacokinetic‐guided Dosing of Efanesoctocog Alfa in Haemophilia A Patients

Haemophilia, EarlyView.
ABSTRACT Introduction Efanesoctocog alfa is a novel factor VIII (FVIII) concentrate with a unique molecular design that enables Von Willebrand Factor‐independent clearance in patients with haemophilia A. Limited sampling strategies (LSSs) are necessary to implement accurate pharmacokinetic (PK)‐guided dosing for efanesoctocog alfa in clinical practice.
Jelien den Hollander, Marjon H. Cnossen, Ron A. A. Mathôt   +2 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

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

florio HAEMO—A Digital Medical Device for Monitoring of Treatment, Symptoms and Physical Activities for People Living With Haemophilia

Haemophilia, EarlyView.
ABSTRACT Introduction Despite therapeutic achievements in haemophilia care, there is still the need to monitor and define personal treatment outcomes and document results to achieve the best possible care. Hence, a need for unbiased, timely and comprehensive real‐world information exists to support informed shared decision‐making regarding treatment ...
Christoph Königs, Jan Astermark, Jan Blatny, Jamie O´Hara, Allfonso Iorio, Claude Negrier, Flora Peyvandi, Katharina Steinitz, Armin J. Reininger, Santiago Bonanad   +9 more
wiley   +1 more source

Clinical and non-clinical aspects of reimbursement policy for orphan drugs in selected European countries. [PDF]

Front Pharmacol
Jakubowski S, Holko P, Nowak R, Warmuth M, Dooms M, Salminen O, Cortial L, Selke GW, Georgi C, Magnússon E, Crisafulli S, Strijbosch F, Mueller T, Grieve E, Danés I, Kawalec P.   +15 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