Results 41 to 50 of about 1,892 (131)

Advanced Therapy Medicinal Products for Eye Diseases: Goals and Challenges

Pharmaceutics, 2021
Advanced therapy medicinal products (ATMPs) are a novel class of medicines with enormous potential to improve treatments for a wide range of diseases, including those affecting eye structures [...]
Yolanda Diebold, Laura García-Posadas
doaj   +1 more source

Phoenix: A Portable, Battery-Powered, and Environmentally Controlled Platform for Long-Distance Transportation of Live-Cell Cultures

Frontiers in Bioengineering and Biotechnology, 2020
Despite the advent of advanced therapy medicinal products (ATMPs) in regenerative medicine, gene therapy, cell therapies, tissue engineering, and immunotherapy, the availability of treatment is limited to patients close to state-of-the-art facilities ...
Brittany N. Willbrand, Sylvia Loh, Caitlin E. O’Connell-Rodwell, Dan O’Connell, Devin M. Ridgley   +4 more
doaj   +1 more source

Current landscape of clinical development and approval of advanced therapies

Molecular Therapy: Methods & Clinical Development, 2021
Advanced therapy medicinal products (ATMPs) are innovative therapies that mainly target orphan diseases and high unmet medical needs. The uncertainty about the product's benefit-risk balance at the time of approval, the limitations of nonclinical ...
Carolina Iglesias-Lopez, Antonia Agustí, Antoni Vallano, Merce Obach   +3 more
doaj   +1 more source

Decorating Carbon Nitride via the Integration of Phosphorylation with Carbon Self‐doping toward Enhanced Photocatalytic Hydrogen Evolution

ENERGY &ENVIRONMENTAL MATERIALS, EarlyView.
The electron transfer from the grafted ATMP to the adjacent heptazine ring can induce the proton transfer of the hydroxyls on ATMP to Pt co‐catalyst nanoparticles. This coupling of proton transfer and electron transport also promotes the separation and transport of charge carriers and therefore advances the performance of photocatalytic hydrogen ...
Yike Li, Zhaohui Wang, Xian Liu, Yongpan Gu, Yongzhu Fu, Zhongjun Li   +5 more
wiley   +1 more source

Impact of Chelators on FAC Following Interaction With NaOCl: A Focused Evidence Synthesis Based on a Systematic Search

Australian Endodontic Journal, EarlyView.
ABSTRACT This systematic review following PRISMA guidelines evaluated the effects of endodontic chelators on free available chlorine (FAC) in sodium hypochlorite (NaOCl), with emphasis on the influence of concentration, contact time, temperature, and pH. Risk of bias was assessed using the Quality Assessment Tool for In Vitro Studies (QUIN).
Hinrich Meijer, Louise Franchina, Poonam Nagarani, Roy George   +3 more
wiley   +1 more source

Decellularised Corneal Stromal Lamina With or Without Autologous Adipose‐Derived Adult Stem Cells for Advanced Keratoconus: 1‐Year Results of a Phase I/IIa Clinical Trial

Clinical &Experimental Ophthalmology, EarlyView.
ABSTRACT Background To report the 1‐year clinical outcomes of decellularised human corneal stromal lamina implantation, with or without autologous adipose‐derived adult stem cells (ADASCs), for advanced keratoconus in a European cohort. Methods In this prospective, randomised Phase I/IIa trial, 10 eyes of 10 patients were allocated to decellularised ...
Hao Zhang, Jorge L. Alió del Barrio, Alejandra E. Rodriguez, Rafael I. Barraquer, Francisco Arnalich‐Montiel, David Mingo‐Botín, María Eugenia Fernández Santos, Jorge L. Alió   +7 more
wiley   +1 more source

Bringing Gene Therapy Into Real World Clinical Practice

Haemophilia, EarlyView.
ABSTRACT Introduction Adeno‐associated virus (AAV)‐based gene therapy for haemophilia has shifted therapeutic paradigms by enabling hepatic gene transfer, restoring endogenous clotting factor expression, and reducing reliance on conventional prophylactic treatments. Two products, valoctocogene roxaparvovec (haemophilia A) and etranacogene dezaparvovec (
Wolfgang Miesbach, Pratima Chowdary, Margareth C. Ozelo   +2 more
wiley   +1 more source

Italian Patients Journey for Gene Therapy in Haemophilia A

Haemophilia, EarlyView.
ABSTRACT Introduction Gene therapy (GT) provides sustained FVIII levels without repeated infusions in Hemophilia A (HA) patients thus overcoming a major limitation of replacement therapy. However, issues remain e.g., patient selection criteria, duration and variability of transgene expression, quality of life and long‐term safety.
Giovanni Di Minno, Gaia Spadarella, Alessandra Borchiellini, Anna Borrelli, Andrea Buzzi, Giancarlo Castaman, Michele Cecchi, Raimondo De Cristofaro, Vincenzo La Mura, Angelo Lupi, Giuseppe Malcangi, Maria Elisa Mancuso, Maria Francesca Mansueto, Cristina Santoro, Rita Carlotta Santoro, Luigi Piero Solimeno, Annarita Tagliaferri, Ezio Zanon, Isabella Garagiola, Flora Peyvandi   +19 more
wiley   +1 more source

Harnessing the Therapeutic Potential of Cell Secretomes and Extracellular Vesicles for Craniofacial Regenerative Applications

Journal of Periodontal Research, EarlyView.
The scoping review summarizes the current preclinical and clinical evidence for the use of “cell‐free” therapies in craniofacial (periodontal, bone and soft‐tissue) regeneration. It also aims to highlight key challenges and strategies towards the clinical translation of these therapies.
Siddharth Shanbhag, Magdalena Mayol, Danijel Domic, Madhusudhan Reddy Bobbili, Johannes Grillari, Mariano Sanz, Reinhard Gruber   +6 more
wiley   +1 more source

Delivery of adoptive cell therapy in the context of the health-care system in the UK: challenges for clinical sites

Therapeutic Advances in Vaccines and Immunotherapy, 2020
Advanced Therapy Medicinal Products (ATMPs) comprise novel cell, tissue and gene therapies and offer the potential of durable remissions for diseases where there is a high unmet clinical need.
Manon Pillai, Michelle M. Davies, Fiona C. Thistlethwaite   +2 more
doaj   +1 more source