CFTR modulators response of S737F and T465N CFTR variants on patient-derived rectal organoids
Orphanet Journal of Rare DiseasesBackground Predictions based on patient-derived materials of CFTR modulators efficacy have been performed lately in patient-derived cells, extending FDA-approved drugs for CF patients harboring rare variants.
Karina Kleinfelder +6 more
doaj +1 more source
Corrin Ring Modification in Peptide Drug Development – a Brief History of “Corrination”
ChemMedChem, EarlyView.This review highlights the applications of vitamin B12 conjugation (“corrination”) to peptides in the field of peptide‐based therapeutics, focusing on improved physio‐chemical properties, targeted/localized drug delivery, and enhanced oral bioavailability.
Nancy Cham, Robert P. Doyle
wiley +1 more source
Italian Journal of PediatricsBackground Multiple Breath washout (MBW) represents an important tool to detect early a possible pulmonary exacerbation especially in Cystic Fibrosis (CF) disease.
Matteo De Marchis +7 more
doaj +1 more source
Sweat Composition in Relation to Rate of Sweating in Patients with Cystic Fibrosis of the Pancreas [PDF]
, 1968 H. M. Emrich +5 more
openalex +1 more source
Methods for the Generation of Single‐Payload Antibody‐Drug Conjugates
ChemMedChem, EarlyView.Antibody‐drug conjugates (ADCs) have emerged as a powerful form of targeted therapeutic. However, despite the myriad methods to generate even‐integer drug‐to‐antibody ratio (DAR) ADCs, strategies to generate DAR 1 are seldom reported despite the property tuning advantages of low drug loading or in enabling access to antibody‐protein constructs.
Thomas Wharton, David R. Spring
wiley +1 more source
Interpretable Graph Convolutional Neural Networks for Inference on Noisy Knowledge Graphs [PDF]
arXiv, 2018 In this work, we provide a new formulation for Graph Convolutional Neural Networks (GCNNs) for link prediction on graph data that addresses common challenges for biomedical knowledge graphs (KGs). We introduce a regularized attention mechanism to GCNNs that not only improves performance on clean datasets, but also favorably accommodates noise in KGs, a
arxiv
A Sodium Transport Inhibitory Factor in the Saliva of Patients with Cystic Fibrosis of the Pancreas [PDF]
, 1967 J. A. Mangos +2 more
openalex +1 more source
Cystic fibrosis of the pancreas: structural changes in peripheral airways [PDF]
, 1968 John R. Esterly, Ella H. Oppenheimer
openalex +1 more source
Impact of Model‐Informed Drug Development on Drug Development Cycle Times and Clinical Trial Cost
Clinical Pharmacology &Therapeutics, EarlyView.Model‐informed drug development (MIDD) integrates data to quantify benefit/risk informing objective drug discovery and development decisions. An additional critical benefit of MIDD is postulated to be improvement in trial and program efficiencies. While the application of MIDD has grown, there have been no clear examples across programs to demonstrate ...
Vaishali Sahasrabudhe +4 more
wiley +1 more source
Systemic therapy‐based split‐course stereotactic body radiation therapy
Precision Radiation Oncology, Volume 6, Issue 4, Page 306-320, December 2022., 2022 Abstract Purpose Given the therapeutic toxicity of concurrent chemoradiotherapy, the dose of chemotherapy is always limited. Hence, the question of how to administer adequate chemotherapy to synchronize stereotactic body radiation therapy (SBRT) treatment strategy for maximizing the benefits of neoadjuvant therapy to improve prognosis is a challenging ...
Rong Zheng +3 more
wiley +1 more source