Simple and economical HandClench Relaxometer device for reliable and sensitive measurement of grip myotonia in myotonic dystrophy.

Grip myotonia and weakness are attractive treatment response biomarkers in clinical trials of myotonic dystrophy type 1 (DM1). There is a need to develop simple, patient-friendly and reproducible methods of quantifying grip myotonia in multisite trial settings. We designed a HandClench Relaxometer (HCR) that measures grip myotonia and strength.

Therapeutic targeting of ATR yields durable regressions in small cell lung cancers with high replication stress.

Small cell neuroendocrine cancers (SCNCs) are recalcitrant cancers arising from diverse primary sites that lack effective treatments. Using chemical genetic screens, we identified inhibition of ataxia telangiectasia and rad3 related (ATR), the primary activator of the replication stress response, and topoisomerase I (TOP1), nuclear enzyme that suppresses genomic instability, as synergistically cytotoxic in small cell lung cancer (SCLC). In a proof-of-concept study, we combined M6620 (berzosertib), first-in-class ATR inhibitor, and TOP1 inhibitor topotecan in patients with relapsed SCNCs.

Respiratory magnetic resonance imaging biomarkers in Duchenne muscular dystrophy.

Objective: To examine the diaphragm and chest wall dynamics with cine breathing magnetic resonance imaging (MRI) in ambulatory boys with Duchenne muscular dystrophy (DMD) without respiratory symptoms and controls.

Methods: In 11 DMD boys and 15 controls, cine MRI of maximal breathing was recorded for 10 sec. The lung segmentations were done by an automated pipeline based on a Holistically-Nested Network model (HNN method).

Skeletal muscle water T as a biomarker of disease status and exercise effects in patients with Duchenne muscular dystrophy.

The purpose of this study was to examine exercise effects on muscle water T in patients with Duchenne muscular dystrophy (DMD). In 12 DMD subjects and 19 controls, lower leg muscle fat (%) was measured by Dixon and muscle water T and R (1/T) by the tri-exponential model. Muscle water R was measured again at 3 hours after an ankle dorsiflexion exercise.

Qualitative comparison of coronary angiograms between 4 French catheters with an advanced cardiovascular injection system and 6 French catheters with manual injection.

Objectives: This study aimed to test whether advanced cardiovascular injection (ACI) via 4 French (Fr) catheters is more advantageous than manual injection with 6 Fr catheters.

Background: For coronary angiography, proponents of contrast media ACI believe it utilizes smaller-diameter catheters when compared with manual injection, without compromising the quality of the angiogram, and has the potential to reduce the amount of contrast used and lessen the use of closure devices.

Methods: In a prospective study, 200 consecutive patients referred for elective coronary angiography were randomized to standard 6 Fr catheters with manual injection versus 4 Fr catheters with ACI.