Results 131 to 140 of about 1,722 (231)

Optimizing Oligonucleotide Therapeutics: A Model‐Informed Drug Development Perspective

Clinical and Translational Science, Volume 19, Issue 5, May 2026.
ABSTRACT Oligonucleotide therapies have emerged as a powerful therapeutic class, providing novel solutions for diverse diseases through antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), and aptamers that specifically target and modulate gene expression or protein function. However, oligonucleotide development faces distinct challenges,
Ye Yuan, Vishnu Sharma, Venkatesh Atul Bhattaram, Xiaolei Pan, Justin Earp, Yun Wang, Jiang Liu, Hao Zhu   +7 more
wiley   +1 more source

Respiratory effects of nusinersen treatment in pediatric patients with spinal muscular atrophy types 2 and 3. [PDF]

Eur J Pediatr
Rochman M, Shay Levanon D, Amirav I, Be'er M, Cahal M, Besor O, Fattal-Valevski A, Lavie M.   +7 more
europepmc   +1 more source

Compound Muscle Action Potential (CMAP) Amplitude Trajectories and Pattern in Adults with 5q-Spinal Muscular Atrophy Receiving Nusinersen Therapy: A Multicenter, Binational Observational Study. [PDF]

Eur J Neurol
Bjelica B, Wohnrade C, Osmanovic A, Schreiber-Katz O, Koerner S, Kollewe K, Haeckl S, Freigang M, Cordts I, Becker B, Vogt C, Muhandes MT, Günther R, Deschauer M, Koch JC, Tuerk M, Regensburger M, Neuwirth C, Petri S.   +18 more
europepmc   +1 more source

Mother tongue instruction as a sticky object: The making of a register of denunciation

Journal of Linguistic Anthropology, Volume 36, Issue 1, May 2026.
Abstract This article examines the making of a political register to denounce mother tongue instruction (MTI) in Sweden. Nationally mandated since 1977, MTI is a state‐sponsored, curriculum‐stipulated subject for minority pupils of over 187 languages other than Swedish.
Scarlett Mannish, Linus Salö
wiley   +1 more source

Comorbidities in spinal muscular atrophy and their impact on the course of the underlying disease: a real-life observational study. [PDF]

BMC Musculoskelet Disord
Błauciak M, Ubysz J, Wieczorek M, Koszewicz M.   +3 more
europepmc   +1 more source

A Personal Exploration of Oral Health in Amyotrophic Lateral Sclerosis (ALS) Through the Eyes of a Multifaceted Authority

Journal of Oral Rehabilitation, Volume 53, Issue 5, Page 1059-1064, May 2026.
This study resulted in agendas to improve oral health care, research, and education, advocating for a shift in ALS oral health care: from neglect to proactive integration. ABSTRACT Background and Objective Amyotrophic Lateral Sclerosis (ALS) is a progressive neurodegenerative disease that impairs motor function, including oral musculature, complicating
Merel C. Verhoeff, Maurits K. A. van Selms, Frank Lobbezoo   +2 more
wiley   +1 more source

Real-world evidence of Nusinersen treatment for patients with spinal muscular atrophy in the Kingdom of Saudi Arabia: Initial insights from the Saudi national spinal muscular atrophy program. [PDF]

Saudi Med J
Al-Jedai AH, Almudaiheem HY, Alaama TY, Alsaman ASS, Abdo Bashiri F, Almuhaizae MA, Alghamdi FA, Alsakran AA, Matter EAY, Ibrahim FZ.   +9 more
europepmc   +1 more source

Harmonic and power spectrum analysis of vertical ground reaction force in patients who have a stroke within 3 months

Experimental Physiology, Volume 111, Issue 5, Page 2528-2542, 1 May 2026.
Abstract Stroke remains a primary cause of disability globally. An in‐depth comprehension of gait impairments following a stroke is vital for crafting effective therapeutic interventions, and vertical ground reaction forces (vGRF) provide valuable insights into these mechanics.
Li Zhang, Hongfang Yao, Shuisheng Fu, Jung Hung Chien   +3 more
wiley   +1 more source

Hirayama Disease in a Young Male: A Case Report. [PDF]

JNMA J Nepal Med Assoc
Paudel S, Rokaha PS, Rokaha PS, Karki L, Thapa P.   +4 more
europepmc   +1 more source

Course of joint range of motion in children with spinal muscular atrophy receiving disease-modifying treatment. [PDF]

Orphanet J Rare Dis
Oude Lansink ILB, Gorter JW, van der Pol WL, Vd Woude DR, Scheiberlich PJ, van Eijk RPA, Bartels B, Beelen A.   +7 more
europepmc   +1 more source