Reduced Sperm Counts in Mice with <i>Dmd</i> Exon 51 Frameshift Mutations. [PDF]
Ilchuk LA +7 more
europepmc +1 more source
Feedback Protocols for Dissipation Control in Small Systems
Single‐molecule pulling experiments recover folding free energies from irreversible work, but dissipation biases the result. Comparing feedback protocols on a model DNA hairpin at equal measurement time, the authors show that reduced dissipation does not always improve free‐energy estimates.
S. Peyriere, F. Ritort
wiley +1 more source
Dynamic changes in total hemoglobin concentration in the frontal region during motion-based video gameplay in patients with Duchenne muscular dystrophy: a pilot study using portable near-infrared spectroscopy (NIRS). [PDF]
Okano S, Mori S, Nishizawa H.
europepmc +1 more source
Abstract Histology is a visually complex and cognitively demanding subject often perceived as abstract, memorization‐heavy, and disconnected from clinical practice. Despite its foundational importance, histology is frequently under‐emphasized relative to gross anatomy and lacks interactive, multimodal resources tailored to its unique challenges.
Anika Knowles +2 more
wiley +1 more source
Influence of community disadvantage on cognitive performance in son-mother dyads in Duchenne muscular dystrophy. [PDF]
Karra H +7 more
europepmc +1 more source
Nucleic Acid Therapeutics in Rare Disease: From Precision Medicine to Programmable Platforms
ABSTRACT Nucleic acid therapeutics (NATs), including antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), messenger RNA (mRNA) therapeutics and programmable editing technologies, are reshaping the therapeutic landscape for inherited metabolic and genetic disease.
Tomasz Z. Tomkiewicz +3 more
wiley +1 more source
Should taurine be pursued further as a supportive nutraceutical for Duchenne muscular dystrophy? Gathering evidence in a zebrafish disease model to confirm or refute the benefits - Stage 1 Report. [PDF]
Vanhove L +8 more
europepmc +1 more source
Clinical History and Burden of Gaucher Disease Type 3: A Cross‐Sectional Multinational Study
ABSTRACT Gaucher disease Type 3 (GD3) is a rare genetic neurodegenerative disorder, characterized by heterogeneity in symptom burden, disease severity, and progression. There is limited evidence on the burden of GD3. This retrospective, cross‐sectional study estimated the burden of illness associated with GD3 using descriptive analyses of clinician ...
Eugen Mengel +10 more
wiley +1 more source

