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Results for “Therapy development”

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At least 19 recordsLinked to original sources
PubMed2026

InsightRP2-An Interdisciplinary Approach Toward Therapy Development in RP2-Associated Retinopathy.

Advancing the development of specific gene therapies for rare genetic eye disorders is a major challenge in modern translational vision research. It requires integrated clinical, molecular, mechanistic, and regulatory expertise, yet these aspects are often addressed in isolation. Combining complementary skills, knowledge, and expertise within an integrated, highly interactive research framework has the capacity to drive and advance therapeutic development. Moreover, early-stage involvement of patients and targeted clinical observation, alongside molecular and therapeutic research, potentially enable clinical trial readiness and support the translation of preclinical therapeutic innovations into clinical care. In this Perspective article, we present the InsightRP2 framework, an integrated translational strategy that incorporates clinical data, artificial intelligence-supported imaging analysis, experimental disease modeling, and adeno-associated virus design with the aim to facilitate the development of a targeted gene therapy for RP2-associated retinitis pigmentosa.

AAV↗
PubMed1979

Structured learning therapy: development and evaluation.

This paper describes the procedures, materials, and evaluation of Structured Learning Therapy, one of several skill training therapies to emerge in recent years. A number of issues are considered in regard to enhanced outcomes for such therapies, particularly means for more successful transfer of newly learned skills from therapy to real-life settings, and prescriptive use of such psycho-educational treatments.

Evaluation Studies as Topic↗
PubMed2026

New approaches to uncover COPD pathobiology and develop therapies.

Chronic obstructive pulmonary disease (COPD) was the third leading cause of global mortality in 2011 but receives limited attention and research funding. This Review describes the current knowledge on COPD risk factors, including genetic and epigenetic determinants and their interactions with the microbiome and environmental exposures. Preclinical models are being refined and single-cell transcriptomic, metabolomic, and proteomic technologies are being implemented to investigate the molecular mechanisms of disease progression. Patient cohorts to define biomarkers of early disease and the latest approaches to diagnose pre-COPD are essential to accelerate the development of novel and effective therapeutic interventions and translate new findings into clinical trials. This Review is a summary of topics covered by a symposium organized by the COPD-iNET consortium, an international network of researchers who have established a platform that facilitates collaboration of this multidisciplinary group of preclinical, translational, and clinical researchers.

Humans↗
PubMed1987

[Effectiveness of isolation ultracentrifugation in the combined treatment of heart failure refractory to therapy developing in the presence of severe arterial hypertension with stable and malignant courses].

The feasibility and practicability of isolated ultrafiltration, or extracorporeal evacuation of excessive body fluid, was assessed in patients with severe stable and malignant arterial hypertension, complicated by refractory heart failure. This procedure, added to combined treatment, is a means to overcome resistance to medication, achieve a stable compensation of circulatory insufficiency symptoms, bring down arterial blood pressure and use smaller doses of hypotensive and diuretic drugs. Isolated ultrafiltration proves less effective in patients with cachectic circulatory insufficiency.

Acute Disease↗
PubMed1989

Observing client-therapist interaction in behaviour therapy: development and first application of an observational system.

A category system for coding the interaction in psychotherapy (CIP) is presented. Six therapists carried out 64 standardized intake sessions with clients presenting with chronic insomnia. The sessions were fully coded by three raters using the CIP. The 19 client categories as well as the 18 therapist categories showed sufficient reliability with kappa coefficients of 0.80 and 0.79 respectively. The frequencies of the client and the therapist categories of the first versus the second half of the interview were compared. Shifts in the behavioural patterns of therapists and clients during the course of the interviews are in accordance with the given rationale of the session. Correlations between the observational data and subjective ratings of the participants are further evidence for the utility of the CIP.

Adult↗
PubMed2025

Screening rare genetic diagnoses for amenability to bespoke antisense oligonucleotide therapy development: A retrospective cohort study.

PURPOSE: To estimate the proportion of molecular genetic diagnoses in a real-world, phenotypically heterogeneous patient cohort that are amenable to antisense oligonucleotide (ASO) treatment. METHODS: We retrospectively applied the N=1 Collaborative's Variant Assessments toward Eligibility for Antisense Oligonucleotide Treatment guidelines to all diagnostic variants found by clinical genome-wide sequencing at a single pediatric hospital in 532 patients over a 6-year period. Variants were classified as either "eligible," "likely eligible," "unlikely eligible," or "not eligible" in relation to the different ASO approaches, or "unable to assess." RESULTS: In total, 25 unique variants across 26 patients (4.9% of 532 patients) were eligible or likely eligible for ASO treatment at a molecular genetic level, via canonical exon skipping (4), splice correction (3), or messenger RNA knockdown (19). Only 8 of these molecular genetic diagnoses were made within a year of symptom onset. After considering disease and delivery related factors, 11 diagnoses were still considered candidates for bespoke ASO development. CONCLUSION: A meaningful proportion of genetic diagnoses identified by genome-wide sequencing may be amenable to ASO treatment. These results underscore the importance of timely diagnosis, and the proactive identification and accelerated functional testing of genetic variants amenable to ASO treatments.

Humans↗
PubMed1989

[New radiopharmaceuticals for oncologic diagnosis and therapy: developments in radioimmunoscintigraphy and radioimmunotherapy].

131I labeled monoclonal antibodies are at a disadvantage for radioimmunoscintigraphy due to the in-vivo cleavage of radioiodine by deiodases. Alternative labeling with 99mTc was not very successful because of the lack of suitable ligands for stable technetium complexes. New developments in this field will be presented using stable 99mTc-N2S2 complexes. Some comments on radioimmunotherapy have also been added.

Antibodies, Monoclonal↗
PubMed1985

Prosthetic valve endocarditis caused by Staphylococcus epidermidis. Development of rifampin resistance during vancomycin and rifampin therapy.

Development of rifampin-resistant Staphylococcus epidermidis was documented in three patients receiving vancomycin and rifampin therapy for prosthetic valve endocarditis. The minimum inhibitory concentrations and minimum bactericidal concentrations of rifampin for the original three isolates were 0.4 micrograms/mL or less. Organisms cultured during relapse or prosthetic valve replacement had minimum inhibitory concentrations of rifampin that were 12.5, 50, and greater than 100 micrograms/mL, respectively. Surgical intervention was necessary in all patients. One died, and one required a second prosthetic valve placement. The patients were treated with vancomycin plus aminoglycoside following identification of rifampin resistance. These observations suggest that vancomycin and rifampin may not be adequate therapy for prosthetic valve endocarditis caused by S epidermidis.

Drug Resistance, Microbial↗
PubMed1977

[Pathology and clinical aspects of retarded child development. Diagnosis and therapy of defective development].

21 cases of "small for date" pregnancies were analysed. Serial ultrasonic tracings and determinations of total estrogen excretion in the 24-hour-urine and of serum HPL concentration were carried out. The fetal heart rate was measured by cardiotocogram. Retrospectively, the patients were divided into 3 groups according to birth weight. All patients were treated with a combination of Complamin, Calciparin and Partusisten. Fetal growth retardation could not be stopped by this treatment; there was, however, definite fetal weight gain following long term therapy. Results of HPL and total estrogen determinations were inconclusive; in most cases, however, a fall of concentrations was observed. Following long term therapy a rise in concentration up to almost normal values was seen. The positive effect of therapy was best shown by serial cardiotograms.

Betamethasone↗
PubMed1991

A checklist to assess patient education in physical therapy practice: development and reliability.

Patient education in physical therapy is gaining attention because it can contribute to patient compliance and prevention. This article describes the development of an assessment tool for investigating patient education in physical therapy. A checklist of 65 educational activity items was constructed. The investigators tested the applicability of the checklist using 227 audiotaped treatment sessions involving patients from various private practices in the Netherlands. The 227 audiotaped sessions covered the entire period of treatment for 25 patients, each of whom participated in an average of 9 treatment sessions. The results showed that all except 1 of the 65 educational activities occurred in the 227 treatment sessions and that no educational activities occurred that could not be rated in the checklist. These findings may indicate that the checklist covers the entire range of educational activities currently applied by physical therapists. The reliability of the checklist was tested using a subsample of the treatments. The average scores for interrater and intrarater reliability, as determined by the use of the Cramer's V coefficient of association for ordinal data, were .76 and .81, respectively. The checklist can be used to investigate the current educational activities in physical therapy practice. In addition, the checklist can be used in peer review and can contribute to the development of standards for the quality of care. [Sluijs EM. A checklist to assess patient education in physical therapy practice: development and reliability.

Educational Measurement↗
PubMed1976

Developing physical therapy fee schedules based on Social Security Amendments of 1972.

Implications of the requirements related to the documentation and justification of the cost of physical therapy services are considered. These implications include 1) the cost components of physical therapy services, 2) the development of fee schedules, and 3) reimbursement requirements of third-party payers. The development of a fee schedule based on a sampling of the prevailing rates in two San Francisco Bay area counties is presented. The relationship between cost accounting, community standards, and the "prevailing rate" as defined by Medicare is documented. Public Law 92-603 and its ramifications for physical therapy fees and salaries are discussed.

California↗