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Accelerating novel therapies for sickle cell disease with RWE: ASH RC Data Hub as a strategically coordinated registry network.

AbstractReal-world evidence (RWE) has been used to improve quality of care, accelerate innovation, and evaluate emerging therapies for drugs, devices, and biologics. The Coordinated Registry Network (CRN), aggregating and linking highly curated patient data, has emerged as a model for RWE generation that has guided the development of the American Society of Hematology Research Collaborative (ASH RC) Data Hub. With the aim of bolstering research, enhancing clinical care, and expediting evidence generation using RWE, the ASH RC and the Innovative Genomics Institute launched a joint initiative, "

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… Members of the work groups included experts from academia, federal agencies, industry, patient organizations, and professional societies. … CRNs offer a unique organizational structure and processes that accelerate innovation more rapidly than industry and government. 27 , 28 , 29 The expertise brought by partners from various backgrounds, including industry, academia, regulatory bodies, health care providers, and patient advocacy groups …

Translation of PET radiotracers for cancer imaging: recommendations from the National Cancer Imaging Translational Accelerator (NCITA) consensus meeting.

BackgroundThe clinical translation of positron emission tomography (PET) radiotracers for cancer management presents complex challenges. We have developed consensus-based recommendations for preclinical and clinical assessment of novel and established radiotracers, applied to image different cancer types, to improve the standardisation of translational methodologies and accelerate clinical implementation.MethodsA consensus process was developed using the RAND/UCLA Appropriateness Method (RAM) to gather insights from a multidisciplinary panel of 38 key stakeholders on the appropriateness of pre

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… Here, we present the findings of a collaborative consensus process, involving a multidisciplinary panel, including stakeholders from academia, industry, UK funding and regulatory bodies, NHS England and patient and public involvement (PPI). … Consensus was agreed on the need for an open access federated repository to facilitate reporting and sharing of clinical imaging data by academia and industry, aligned with the FAIR principles (data must be Findable, Accessible, Interoperable and Reusable) [ 32 ]. …

Regulatory insights on gene therapies, adeno-associated virus-based gene therapies, cell/tissue-based products, and medical care/practice in cell and gene therapies: Report from the 7th Asia partnership conference - April 25, 2024.

The 7th Asia Partnership Conference of Regenerative Medicine (APACRM) was held both in person and online on April 25, 2024, to promote the regulatory harmonization of regenerative medicine products across Asia. Recognizing domestic regulatory guidelines and their underlying rationales within each country and region is an important initial step toward achieving harmonious regulation. The 7th APACRM featured open dialogues on the non-clinical evaluations of adeno-associated virus gene therapy products, regulation of gene therapies, considerations for conducting clinical trials, and provision of

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… In Japan, startups and academia are the key drivers of innovation in regenerative medicine, often creating initial product concepts. Japan excels in basic and early clinical research, which is vital for successful product commercialization. … Taiwan Chia-Ning Shen, a research fellow/Professor at the Genomics Research Center, Academia Sinica on behalf of the BPIPO, presented the status of cell therapy and RM in Taiwan. …

A Comprehensive Review of Interventional Clinical Trials in Patients with Bone Metastases.

Bone metastasis is one of the most important factors associated with poor prognosis for patients with prostate, breast, thyroid, and lung cancer. In the past two decades, 651 clinical trials, including 554 interventional trials, were being registered in ClinicalTrials.gov and pharma.id.informa.com to combat bone metastases from different perspectives. In this review, we comprehensively analyzed, regrouped, and discussed all the interventional trials on bone metastases. Clinical trials were re-grouped into bone-targeting agents, radiotherapy, small molecule targeted therapy, combination therapy

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… The clinical trials were sponsored by academia (252, 40.7%), industry (198, 32%), and the collaboration of industry and academia (169, 27.3%) ( Figure 2A ). Of the 619 clinical trials, 569 had country information. … To date, over 100,000 compounds have been screened in more than 1000 cancer cell lines by many different institutes either in academia or in industries, such as NCI, the Sanger Institute, the Broad Institute, the Institute for Molecular Medicine Finland, GlaxoSmithKline, the MGH cancer center, Novartis …

Выдержка гемпта (Extractum Cannabis) в деле " Лечение кишечных расстройств и диспепсии: исторические сведения из Барселоны, Испания " .

В этом исследовании изучается траектория интереса и использования экстракта каннабиса (выдержка канабиса сативы L.) для симптоматического лечения легких желудочно-кишечных расстройств и диспепсии в Барселоне XIX и начала XX века (Каталония, Испания) 1939, на основе обзора первичных источников. Цель настоящего документа заключается в том, чтобы представить исторический фармацевтический и прикладный обзор использования гематома (Cannabis L.) в медицинских целях до его запрещения, способствуя тем самым его признанию в качестве медицинского продукта. Представленная информация свидетельствует об ис

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… Background: Historical Lacunae and Geographical Imbalance in the Documentation of Medicinal Hemp The dominance of English in scientific publishing and academia is known to pose some challenges to research and the sharing of knowledge produced in other languages [ 7 , 8 , 9 ]. … Administración (Alcalá de Henares), Archivo Histórico Nacional (Madrid), Arxiu Històric & Arxiu Municipal Contemporani (Barcelona), Arxiu Nacional de Catalunya (Sant Cugat del Vallès), Biblioteca de Catalunya (Barcelona), Biblioteca Pública Arús (Barcelona), Oficina Española de Patentes y Marcas (Madrid), Real Academia …

Сайентометрический анализ неврологических публикаций в Иране.

История вопроса: Несомненно, медицина родилась с самого начала сотворения человека. Одной из его важных ветвей является неврология. Нейрофизики в Иране с небольшой задержкой из первого мира, с усилиями исследователей, открыли путь к диагностике и лечению неврологических заболеваний, и мы достигли того места, где мы находимся. Методы: В этом библиометрическом и сайентометрическом исследовании мы провели оценку процесса неврологии в Иране. Сославшись на надежные индексы, мы проверили Web of Science (WOS), PubMed и Scopus начиная с 1963 года. Мы показали опубликованную деятельность иранцев в виде

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… topics (first 50 topics) in Iranian neurological research Discussion This study reveals that clinical neurologists in Iran have contributed to over 3% of the country's total medical publications in international journals ‒ a notable figure given that neurology faculty represent only ~1% of medical academia … The disproportionate research productivity of Iran's neurology faculty ‒ comprising just 1.2% of medical academia yet contributing to 9.7% of publications ‒ presents important implications for research evaluation. 15 While international indices show strong growth ( Figure 3 ), significant limitations …

Peripheral Nervous, Hepatic, and Gastrointestinal Endpoints for AL Amyloidosis Clinical Trials: Report from the Amyloidosis Forum Multi-organ System Working Group.

Systemic immunoglobulin light chain (AL) amyloidosis is a heterogeneous rare disease driven by a destructive monoclonal gammopathy and typified by misfolded immunoglobulin light and/or heavy chains which aggregate and deposit in organs as insoluble amyloid fibrils. Disease heterogeneity is driven by the degree of multi-systemic involvement; cardiac, renal, neurological, and gastrointestinal (GI) systems are affected to varying degrees in different patients. While prognosis is primarily driven by hematologic response to treatment and outcomes associated with cardiac events and overall survival,

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… The Amyloidosis Forum hosts meetings, workshops, and other scientific activities in the precompetitive domain to bring together representatives from academia, industry, and regulatory agencies, complemented by patient perspectives. … will develop and evaluate candidate composite endpoints and potential surrogate endpoints to facilitate drug development in AL amyloidosis The Multi-organ System Working Group comprised a chairperson (MLM), patient representative, two statisticians, and a panel of AL amyloidosis experts representing academia …

The Amyloidosis Forum: a public private partnership to advance drug development in AL amyloidosis.

BACKGROUND: Immunoglobulin light chain (AL) amyloidosis is a rare, multi-systemic disorder characterized by two disease processes: an underlying plasma cell dyscrasia that provides the source of pathologic light chains, and the resulting organ dysfunction caused by deposition of amyloid light chain fibrils. There are no FDA approved treatments for AL amyloidosis; regimens developed for multiple myeloma are used off-label to treat the plasma cell disorder and no therapies are directed at organ deposition. Thus, an unmet medical need persists despite advances in disease management. A public-priv

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… The PPP seeks to leverage expertise and resources of all stakeholders (academia, industry, patients, and regulatory agencies) for the conduct of mutually beneficial scientific activities in the precompetitive domain to support bringing new, safe and efficacious therapies to patients with AL amyloidosis … The PPP seeks to leverage expertise and resources of all stakeholders (academia, industry, patients, and regulatory agencies) for the conduct of mutually beneficial scientific activities in the precompetitive domain to support bringing new, safe and efficacious therapies to patients with AL Amyloidosis …

Current Trends and Challenges in the Clinical Translation of Nanoparticulate Nanomedicines: Pathways for Translational Development and Commercialization.

The use of nanotechnology in medicine has the potential to have a major impact on human health for the prevention, diagnosis, and treatment of diseases. One particular aspect of the nanomedicine field which has received a great deal of attention is the design and development of nanoparticulate nanomedicines (NNMs) for drug delivery (i.e., drug-containing nanoparticles). NNMs are intended to deliver drugs via various mechanisms: solubilization, passive targeting, active targeting, and triggered release. The NNM approach aims to increase therapeutic efficacy, decrease the therapeutically effecti

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… NNMs for triggered release A third targeting strategy based on stimuli-responsive NNMs, referred to as triggered drug release, is currently receiving much attention from academia and industry. … This will need to involve experts from academia and industry with specialty in pharmaceutics, engineering, biology, medicine, and toxicology. …

Interventions for preventing dengue: a mapping review.

BACKGROUND: Dengue remains a pressing global health challenge, particularly in low- and middle-income countries (LMIC). Despite a growing vo … s, a Brazilian non-governmental organisation, that aims to build bridges between public management, academia, and civil society, to conduct stakeholder engagement. MAIN RESULTS: The searches identified 20,355 records. After deduplication, we screened 16,468 titles and abstracts, then assessed 720 full-text articles for further eligibility. In total, we in …