Results 181 to 190 of about 88,273 (287)

Establishment of a value assessment framework for orphan drugs in China: an application of the discrete choice experiment in multicriteria decision analysis. [PDF]

Front Pharmacol
Zhao Z, Tang X, Hu M.
europepmc   +1 more source

Criteria to define rare diseases and orphan drugs: a systematic review protocol. [PDF]

BMJ Open, 2022
Abozaid GM, Kerr K, McKnight A, Al-Omar HA.   +3 more
europepmc   +1 more source

A Psychometric Analysis of the Haemophilia Quality of Life Questionnaire for Adults Physical Health Subscale and Total Scores: Data From the Phase 3 XTEND‐1 Trial in Patients With Severe Haemophilia A

Haemophilia, EarlyView.
ABSTRACT Introduction Bleeding episodes can impair physical functioning and cause chronic pain in haemophilia A. The Haemophilia Quality of Life Questionnaire for Adults (Haem‐A‐QoL) was administered in XTEND‐1; the Physical Health (PH) subscale evaluated Physical Health/Functioning. Aim To evaluate psychometric properties of Haem‐A‐QoL PH and estimate
Jérôme Msihid, Alfonso Iorio, Amanda Wilson, Nana Kragh, Lauren Nelson, Shanshan Qin, Annemieke Willemze   +6 more
wiley   +1 more source

New opportunities for accessing promising non-oncological orphan drugs. [PDF]

Lancet Reg Health Eur
Hollak CEM, Reparon-Schuijt CC, Verdeyen K, Deesker LJ, Groothoff JW, Vos JMI, Huisman AH, van der Flier S, Pisters-van Roy AAMG.   +8 more
europepmc   +1 more source

Predicting Recovery After Joint Bleeding in Persons With Bleeding Disorders

Haemophilia, EarlyView.
ABSTRACT Introduction Joint bleeds are burdensome and recovery differs from bleed to bleed. Identifying predictors of recovery could enable personalized treatment and monitoring; aiming to prevent long‐term joint deterioration, and facilitate faster return to activities. Aim To identify predictors of time to recovery after joint bleeding in people with
Gijs Aertssen, Lize F. D. van Vulpen, Wouter Foppen, Flora H. P. van Leeuwen, Merel A. Timmer   +4 more
wiley   +1 more source

The clinical assessment of studies on orphan drugs in relation to the EMA's authorization marketing decisions in Europe. [PDF]

Front Pharmacol
Jakubowski S, Malinowski KP, Kawalec P.
europepmc   +1 more source

Prices of Orphan Drugs in Four Western European Countries Before and After Market Exclusivity Expiry: A Cross-Country Comparison of List Prices and Purchase Prices. [PDF]

Appl Health Econ Health Policy, 2023
Dane A, Klein Gebbink AS, Brugma JD, Degrassat-Théas A, Hug MJ, Houlind MB, Paubel P, van der Kuy PHM, Uyl-de Groot CA.   +8 more
europepmc   +1 more source

Treatment With Valoctocogene Roxaparvovec in a Patient With Severe Hemophilia A Led to Sustained Normal FVIII Levels

Haemophilia, EarlyView.
ABSTRACT Introduction Valoctocogene roxaparvovec, an adeno‐associated virus (AAV)‐based gene therapy, enables endogenous factor VIII (FVIII) expression in patients with severe hemophilia A without the need for regular FVIII infusions. Long‐term follow‐up assesses durability, safety, and immune‐related challenges following gene therapy.
Kerstin Herbst, Behnaz Pezeshkpoor, Claudia Klein, Thilo Albert, Philipp Lutz, Christian Strassburg, Ulrich Spengler, Georg Goldmann, Johannes Oldenburg   +8 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

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