Results 111 to 120 of about 68,732 (285)

Regulating Rare Disease: Safely Facilitating Access to Orphan Drugs [PDF]

, 2018
While approximately one in ten Americans suffers from a rare disease, only 5 percent of rare diseases have a U.S. Food and Drug Administration (FDA) approved treatment.
Bannister, Julien B.
core   +1 more source

Areas of research priorities in epilepsy: A position paper of the European Reference Network for Rare and Complex Epilepsies, EpiCARE

Epilepsia Open, EarlyView.
Abstract Objective To define and articulate research priorities in epilepsy identified by the European Reference Network for Rare and Complex Epilepsies (ERN EpiCARE), addressing key unmet needs across the spectrum of rare and complex epilepsies. Methods This position paper was developed through a structured collaborative process involving patient ...
Sébile Tchaicha, Stéphane Auvin, Sándor Beniczky, Andreas Brunklaus, Lieven Lagae, Emilio Perucca, Rainer Surges, Sophie Adler, Christoph Helmstaedter, Floor Jansen, Guido Rubboli, Philippe Ryvlin, Nicola Specchio, Eugen Trinka, Ingmar Blümcke, Valentina de Giorgis, Katarzyna Kotulska, Gaetan Lesca, Masa Malenica, Amy McTague, Rima Nabbout, Rikke Steensbjerre Møller, Sandra Silva Arrieta, Isabella Brambilla, Małgorzata Kosla, Kees Braun, J. Helen Cross, Alexis Arzimanoglou   +27 more
wiley   +1 more source

Healing Beams: Radiation and Radiotherapy in Novels, Poems, Music, Film, Painting

Journal of Medical Radiation Sciences, EarlyView.
Radiation and radiotherapy are perceived by patients and the general public as mysterious, dangerous and healing. The image of radiation and radiotherapy can be analysed by studying novels, poems, music, film and painting. Our paper reviews how radiation and radiotherapy are perceived by patients, the general public, as reflected in various art genres.
Ad A. Kaptein, Jan W. Schoones, Yvette M. van der Linden, Brian M. Hughes   +3 more
wiley   +1 more source

Long term management of patients with cryopyrin-associated periodic syndromes (CAPS): focus on rilonacept (IL-1 Trap)

Biologics: Targets & Therapy, 2008
Leigh D Church1, Sinisa Savic2, Michael F McDermott21Department of Rheumatology, Division of Immunity, Infection and Inflammation, Institute for Biomedical Research, The University of Birmingham, Birmingham, UK; 2Section of Musculoskeletal Disease, Leeds
Leigh D Church, Sinisa Savic, Michael F McDermott   +2 more
doaj  

Intellectual Property & External Consumption Effects: Generalizations from Pharmaceutical Markets [PDF]

There is a long-standing literature that recognizes that an efficient solution in correcting a consumption externality is through applying subsidies and taxes that line up private incentives with social ones.
Stephane Mechoulan, Tomas Philipson
core  

Reshaping study design for faster extrapolation‐based drug approval in pediatric inflammatory bowel diseases: An ESPGHAN–NASPGHAN position paper

Journal of Pediatric Gastroenterology and Nutrition, EarlyView.
Abstract Children with inflammatory bowel diseases (IBD) have limited access to the available advanced therapies, given the lengthy gap between adult and pediatric approval. We aimed to review key hurdles for pediatric trials and recommend practical solutions.
Dan Turner, Amit Assa, Mikkel Malham, Shira Yuval Bar‐Asher, Carla Rayan, Jeffrey S. Hyams, Holm H. Uhlig, Brad Pasternak, David C. Wilson, Hilary K. Michel, Lissy de Ridder, Eric Zuckerman, Marina Aloi, Peter Szitanyi, Marla C. Dubinsky, Anne M. Griffiths, Roberto Saldana, Javier Martín‐de‐Carpi, Eytan Wine, Richard K. Russell, Jeremy Adler, Joseph Picoraro   +21 more
wiley   +1 more source

Recurrent Respiratory Papillomatosis Foundation Position Statement on the Management of Adults With RRP

The Laryngoscope, EarlyView.
ABSTRACT Objective With regulatory approval of HPV‐specific immunotherapy for recurrent respiratory papillomatosis (RRP) and growing experience with systemic bevacizumab, a management algorithm incorporating these medical treatments is warranted. Data Sources and Methods RRP Foundation (RRPF) Key Opinion Leaders offer a proposed management algorithm ...
Simon R. Best, Aaron D. Friedman, Clark A. Rosen, Robert T. Sataloff, Laura A. Matrka, H. Steven Sims, David E. Rosow, Nabil F. Saba, David G. Lott, Adam M. Klein, Ted Mau, Milan R. Amin, Kathryn A. Wikenheiser‐Brokamp, Scott M. Norberg, Kim McClellan, Geoffrey D. Young, Clint T. Allen, on behalf of the Recurrent Respiratory Papillomatosis Foundation   +17 more
wiley   +1 more source

Access to Orphan Medicinal Products in Bulgaria: An Analysis of the Positive Drug List and Individual Access Schemes

Healthcare
Background/Objectives: Orphan medicinal products offer essential treatments for rare diseases, but patient access varies across European Union countries despite a common regulatory framework. In Bulgaria, access is primarily through inclusion in the positive drug list following health technology assessment or via individual access schemes under ...
Rumen Stefanov, Ralitsa Raycheva, Kostadin Kostadinov, Georgi Stefanov, Iva Zdravkova-Aneva, Elizabet Dzhambazova, Georgi Iskrov   +6 more
openaire   +2 more sources

Considerations for Initiation and Maintenance of Foslevodopa/Foscarbidopa for Advanced Parkinson's Disease

Movement Disorders Clinical Practice, EarlyView.
Abstract Background As Parkinson's disease (PD) progresses, motor fluctuations become increasingly difficult to manage with oral medications. Foslevodopa/foscarbidopa (LDp/CDp), delivered as a continuous 24‐h/day subcutaneous infusion, offers continuous levodopa delivery and stable plasma levodopa levels that reduce motor fluctuations. LDp/CDp has been
K. Ray Chaudhuri, Bruno Bergmans, Eric Freire‐Alvarez, Lucie Hopes, Drew S. Kern, Robert S. Kirsner, Giulia Lazzeri, Pedro Mendes‐Bastos, Noriko Nishikawa, Per Odin, Karolina Poplawska‐Domaszewicz, Rajesh Pahwa, Yuval Ramot, Klaus Seppi, Tobias Warnecke, Robert Wiggins, Megha B. Shah, Pavnit Kukreja, Bjoern Fritz, Koray Onuk, Stuart H. Isaacson   +20 more
wiley   +1 more source

Rheumatological complaints in H syndrome: from inflammatory profiling to target treatment in a case study

Pediatric Rheumatology Online Journal
Background H Syndrome is a rare genetic condition caused by biallelic pathogenic variants in the SLC29A3 gene. It is characterized by a wide range of clinical manifestations, many of which are related to the immune-rheumatological field.
Alessandra Tesser, Erica Valencic, Valentina Boz, Gianluca Tornese, Serena Pastore, Manuela Zanatta, Alberto Tommasini   +6 more
doaj   +1 more source