Results 121 to 130 of about 88,754 (289)

Therapeutic Gene Editing: DNA Repair Pathways, Emerging Editors, and Clinical Progress

iNew Medicine, EarlyView.
ABSTRACT The field of gene editing has evolved rapidly over the past decade, progressing from programmable zinc‐finger nucleases (ZFNs) and transcription activator‐like effector nucleases (TALENs) to the widespread adoption of CRISPR‐Cas systems. First‐generation editors catalyzed genome engineering by introducing targeted double‐strand breaks (DSBs ...
Li‐Kuang Tsai, Youcai Xiong, Dongqi Liu, Dongshan Yang, Jifeng Zhang, Jie Xu, Renzhi Han, Y. Eugene Chen   +7 more
wiley   +1 more source

Regulating Innovative Medicine: Fitting Square Pegs in Round Holes [PDF]

, 2005
Increasingly, innovative medical products are creating a quandary for the Food and Drug Administration ( FDA ) because they often transcend the FDA\u27s traditional categorical approach to regulating medical products. In a recent attempt to simplify this
Lavender, Mark
core   +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

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

The gulf between rhetoric & reality : an examination of the gap between development theory and development practice in the care of Cambodian orphans : a thesis presented in partial fulfilment of the requirements for the degree of Master of Philosophy in Development Studies at Massey University, Palmerston North Campus [PDF]

, 2005
Like many other Developing World countries. Cambodia is in the midst of an orphan crisis. At least 77,000 children have lost their parents to AIDS and many thousands more have been orphaned by civil war. landmines and other tragedies.
Greenfield, Craig
core  

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

Analysis of marketed orphan drugs in China. [PDF]

Intractable Rare Dis Res, 2023
Zhi W, Liu M, Yang D, Zhang S, Lu Y, Han J.   +5 more
europepmc   +1 more source

Clinical development and marketing application review times for novel orphan-designated drugs

Frontiers in Medicine
Development of an orphan-designated drug has been more challenging and financially less attractive than that of other drugs due to low prevalence of the condition, poorly defined biomarkers and lack of experience of healthcare providers in diagnosing and
Ebru Demirci, Jennifer Knicley, Lori Fiorentino   +2 more
doaj   +1 more source

Machine Learning for Predictive Modeling in Nanomedicine‐Based Cancer Drug Delivery

Med Research, EarlyView.
The integration of AI/ML into nanomedicine offers a transformative approach to therapeutic design and optimization. Unlike conventional empirical methods, AI/ML models (such as classification, regression, and neural networks) enable the analysis of complex clinical and formulation datasets to predict optimal nanoparticle characteristics and therapeutic
Rohan Chand Sahu, Sanchit Arora, Dinesh Kumar, Ashish Kumar Agrawal   +3 more
wiley   +1 more source