Results 201 to 210 of about 25,450 (266)

Maternal‐Fetal Administration of Risdiplam Partially Rescues the SMNΔ7 Mouse Model of Spinal Muscular Atrophy

Annals of Neurology, EarlyView.
Objective Spinal muscular atrophy (SMA) is caused by deletions or mutations in the survival motor neuron 1 (SMN1) gene and subsequent reduction in the expression of survival motor neuron (SMN) protein. The disease is characterized by degeneration of α motor neurons and subsequent muscle atrophy.
Emma R. Sutton, Ariane Beauvais, Rebecca Yaworski, Hugh J. McMillan, Rashmi Kothary   +4 more
wiley   +1 more source

Neurotransmitter‐Defined Degeneration Patterns in Sporadic and C9orf72‐Associated Amyotrophic Lateral Sclerosis: Predilection to GABAergic, Serotonergic, Opioid, Glutamatergic, Endocannabinoid, and Microglial Systems—Implications for Therapy Development

Annals of Neurology, EarlyView.
Objective Amyotrophic lateral sclerosis (ALS) has a markedly distinctive clinical and neuroradiological signature, with the preferential involvement of specific brain networks and the apparent sparing of others. The molecular underpinnings of the strikingly selective anatomical vulnerability have not been fully elucidated to date despite the potential ...
Marlene Tahedl, We Fong Siah, Jasmin Lope, Jennifer C. Hengeveld, Mark A. Doherty, Russell L. McLaughlin, Orla Hardiman, Ee Ling Tan, Alison Toomey, Jana Kleinerova, Peter Bede   +10 more
wiley   +1 more source

External controls for rare disease drug development: Lessons for emerging and advanced therapeutic modalities. [PDF]

Mol Ther Adv
Hughes SH, Beretich LA, Fuller M, Goodspeed K, Penn M, Valentino LA, McCombs C.   +6 more
europepmc   +1 more source

Therapeutic Applications of Stimuli‐Based Release and Engineering of Extracellular Vesicles

Advanced NanoBiomed Research, EarlyView.
This review summarizes the effects of endogenous and exogenous stimuli, their effects on the natural release of extracellular vesicles, as well as their uptake and release. It also gives an overview of stimuli‐responsive EVs and their therapeutic applications. Extracellular vesicles (EVs), nano‐ to microsized lipid bilayer membrane‐bound particles, are
Gloria Kemunto, Kristen Dellinger
wiley   +1 more source

Rewriting Tumor Immunity With Bacterial Membrane Vesicles: A Versatile and Potent Therapeutic Strategy

Advanced NanoBiomed Research, EarlyView.
Bacterial membrane vesicles (BMVs) emerge as promising platforms for cancer immunotherapy owing to their intrinsic adjuvant properties and tunable cargo delivery capabilities. Their ability to modulate the tumor microenvironment, enhance antitumor immune responses, and support personalized therapeutic strategies highlights their growing potential as ...
Md Sifat Rahi, Md Mijanur Rahman, Asma Talukder, Urooj Yousaf Virk, Jennifer C. Wilson, Nigel McMillan, Ming Q. Wei   +6 more
wiley   +1 more source

Toward Trial Readiness in Congenital Myotonic Dystrophy: A Longitudinal Cohort Study of Predictors of Motor Function in Childhood. [PDF]

Neurol Genet
Kiefer M, Hartman JM, Berggren KN, Butler A, Jones AS, McIntyre M, Hung M, Carrell S, Hale MA, Campbell C, Sansone VA, Johnson NE.   +11 more
europepmc   +1 more source

Nanostructured Deep Eutectic Systems in Healthcare: From Bioactive Solvents to Intelligent Biointerfaces, Wearables, and AI‐Driven Design

Advanced NanoBiomed Research, EarlyView.
Beyond the green solvent paradigm, this review redefines Deep Eutectic Systems (DES) as programmable supramolecular nanoassemblies. We survey their biomedical convergence: stabilizing thermolabile mRNA to enable cold chain‐free logistics, reshaping transdermal microneedle delivery, enabling long‐term wearables via eutectogels, and utilizing Generative ...
Jeesu Moon, Min Seo Kim, Jae‐Seung Lee
wiley   +1 more source

The Missing Link in Ferrocenophane Chemistry: Isolation of a Carba[1]Ferrocenophane

Angewandte Chemie International Edition, EarlyView.
The synthesis and isolation of a crystalline carba[1]ferrocenophane is reported. A single carbon atom is the smallest bridging motif yet incorporated into a ferrocenophane, resulting in a dihedral angle between the cyclopentadienyl ring planes of 36.7°, which is the highest angular deflection from ferrocene's coplanarity observed so far.
Aylin Feuerstein, Sergi Danés, Kathrin Kolling, Bernd Morgenstern, Markus Gallei, André Schäfer   +5 more
wiley   +1 more source

Clinical Experience of Timing Treatment in Newborns with Spinal Muscular Atrophy: A Call for Standardized Screening Practices in Italy. [PDF]

Int J Neonatal Screen
Bitetti I, Iannaccone R, Margiotta G, Varone A.   +3 more
europepmc   +1 more source

Advancements in CO2 Capture Technologies: A Review of Amine Absorption and Alternative Methods for Post‐Combustion Integration

Asia-Pacific Journal of Chemical Engineering, EarlyView.
ABSTRACT Carbon capture technologies are poised to become one of the primary focuses regarding the achievement of net‐zero emissions by the year 2050. In these technologies, particular attention is given to post‐combustion CO2 capture, which is more easily implemented at existing power stations and other industries.
Satyajit Mukherjee, Ashish Kumar, Mayurkumar P. Patil, Rohit B. Meshram   +3 more
wiley   +1 more source