Results 81 to 90 of about 88,273 (287)

Impact of the therapeutic positioning report in the P&R process in Spain: analysis of orphan drugs approved by the European Commission and reimbursed in Spain from 2003 to 2019

Orphanet Journal of Rare Diseases, 2020
Background Pricing and reimbursement decisions for orphan drugs are faced with differences access between European countries depending on each reimbursement policies, evaluation processes and timings.
Xavier Badia, Tania Vico, John Shepherd, Alicia Gil, José Luis Poveda-Andrés, César Hernández   +5 more
doaj   +1 more source

Understanding Disease Heterogeneity and Patient Characteristics in Patients with Amyotrophic Lateral Sclerosis (ALS) [PDF]

, 2019
Background: Amytrophic lateral sclerosis (ALS) is a fatal neurologic disease that is projected to double in worldwide incidence in the next 20 years.
Mathew, Veena
core  

Developing Methodology for the Creation of Clinical Practice Guidelines for Rare Diseases : A Report from RARE-Bestpractices [PDF]

, 2015
Date of Acceptance: 29/05/2015 The research leading to these results has received funding from the (FP7/2007–2013), under grant agreement n 305690 (RARE-Bestpractices project). The opinions, presented here reflect only the authors’ views.
RARE-Bestpractices Consortium
core   +1 more source

A Systematic Review of Adverse Childhood Experiences and Epigenetic Age Acceleration in Later Adult Life Measured With Second and Third‐Generation Epigenetic Clocks

American Journal of Medical Genetics Part B: Neuropsychiatric Genetics, EarlyView.
ABSTRACT We examined the relationship between adverse childhood experiences (ACEs) and epigenetic age acceleration (EAA) in adulthood as measured by second and third generation epigenetic clocks by performing a systematic review of the literature. The electronic databases MEDLINE and EMBASE were searched on 17 July 2023.
Matthew Green, Laura Horsfall, Azam Saied   +2 more
wiley   +1 more source

Drugs for exceptionally rare diseases: a commentary on Hughes et al [PDF]

Recently in this journal, Hughes and colleagues discussed special funding status to ultra-orphan drugs. They concluded that there should be a uniform policy for the provision of orphan drugs across Europe; that complete restriction was impractical, and ...
Claxton, K., McCabe, C., Raftery, J., Tsuchiya, A.   +3 more
core  

Descriptive Epidemiology From the Myhre Syndrome Foundation Registry: The Value of Self‐Reported Data

American Journal of Medical Genetics Part C: Seminars in Medical Genetics, EarlyView.
ABSTRACT Myhre syndrome is an ultrarare genetic disease characterized by short stature, distinct craniofacial features, cardiovascular and respiratory fibrosis and stenosis, neurodevelopmental delays, autism, intellectual disability, and hearing loss. The natural history of Myhre syndrome is still not fully understood due to a small patient population ...
Mary K. Young, Armelle Pindon, Maggie R. Brand, Kate Wears, Katherine H. Young, Alyssa Mendel, Michael J. Lyons   +6 more
wiley   +1 more source

Prediction and classification for GPCR sequences based on ligand specific features [PDF]

, 2006
Functional identification of G-Protein Coupled Receptors (GPCRs) is one of the current focus areas of pharmaceutical research. Although thousands of GPCR sequences are known, many of them are orphan sequences (the activating ligand is unknown). Therefore,
F. Horn, G.E. Tusnády, K.R. Sreekumar, M. Bouvier, R. Karchin, S. Altshul, T. Gudermann, W. Pearson, Y. Huang   +8 more
core   +1 more source

Caregiver Reports on the Needs and Experiences of Children Impacted by Parental Incarceration: Results From an Australian Survey

Australian Journal of Social Issues, EarlyView.
ABSTRACT Children experiencing parental imprisonment are known to be among the most overlooked in our community. They often experience multiple and compounding disadvantages, with long‐term consequences, but receive no specialised assistance. Knowledge about these children and their families is lacking in Australia and is required to inform policy ...
Catherine Flynn, Lana Battaglia, Lorana Bartels, Susan Dennison, Susy Harrigan, Lee Huggins, Sofia Grage‐Moore   +6 more
wiley   +1 more source

Preisregulierung durch Nutzenbewertung bei seltenen Erkrankungen

Monitor Versorgungsforschung
Ziel der Studie ist eine genaue Untersuchung der populationsnormalisierten Behandlungskosten von Orphan Drugs. Zu diesem Zweck wurde bereits im Jahr 2022 eine Studie zur Betrachtung der Therapiekosten von Orphan Drugs für die Jahre 2005 bis 2021 ...
Lukas Maag MSc, Dr. Ariane Höer, Dr. rer. nat. Gerrit Müller   +2 more
doaj   +1 more source

Reimbursement Status and Recommendations Related to Orphan Drugs in European Countries

Frontiers in Pharmacology, 2019
Objective: To review the reimbursement recommendations issued by selected European health technology assessment agencies for orphan drugs and the reimbursement status of these drugs; to assess the relationship between the type of recommendation and ...
Ewa Stawowczyk, Krzysztof Piotr Malinowski, Paweł Kawalec, Rafał Bobiński, Jacek Siwiec, Dimitra Panteli, Dimitra Panteli, Dimitra Panteli, Helene Eckhardt, Helene Eckhardt, Helene Eckhardt, Steven Simoens, Antònia Agusti, Antònia Agusti, Marc Dooms, Andrzej Pilc, Andrzej Pilc   +16 more
doaj   +1 more source