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United States and European Union laws demand separate clinical studies in children as a condition for drugs' marketing approval. Justified by carefully framed pseudo-scientific wordings, more so the European Medicines Agency than the United States Food and Drug Administration, "Pediatric Drug Development" is probably the largest abuse in medical research in history. Preterm newborns are immature and vulnerable, but they grow.
View Article and Find Full Text PDFSarcolemma resilience and skeletal muscle health require O-mannosylation of dystroglycan.
Skelet Muscle
January 2025
Department of Molecular Physiology and Biophysics, and Department of Neurology, Howard Hughes Medical Institute, Senator Paul D. Wellstone Muscular Dystrophy Specialized Research Center, Roy J. and Lucille A. Carver College of Medicine, The University of Iowa, Iowa City, Iowa, USA.
Background: Maintaining the connection between skeletal muscle fibers and the surrounding basement membrane is essential for muscle function. Dystroglycan (DG) serves as a basement membrane extracellular matrix (ECM) receptor in many cells, and is also expressed in the outward-facing membrane, or sarcolemma, of skeletal muscle fibers. DG is a transmembrane protein comprised of two subunits: alpha-DG (α-DG), which resides in the peripheral membrane, and beta-DG (β-DG), which spans the membrane to intracellular regions.
View Article and Find Full Text PDFFavourable Outcome of Relapsed PCNSL Among Transplant Eligble Patients.
Eur J Haematol
January 2025
Institute of Clinical Medicine, Oncology, University of Eastern Finland, Kuopio, Finland.
Purpose: The prognosis of relapsed primary central nervous system lymphoma remains a concern. This study aimed to compare the effects of various patient- and disease-related factors on the prognosis of relapsed primary central nervous system lymphoma (PCNSL).
Methods: We retrospectively collected real-world data from eight Finnish hospitals on 198 patients diagnosed with PCNSL between 2003 and 2020.
Efficacy of tranilast in preventing exacerbating cardiac function and death from heart failure in muscular dystrophy patients with advanced-stage heart failure: a single-arm, open-label, multicenter study.
Orphanet J Rare Dis
January 2025
Department of Cardiac Physiology, National Cerebral and Cardiovascular Center Research Institute, 6-1 Kishibe-Shimmachi, Suita, Osaka, 564-8565, Japan.
Background: Transient receptor potential cation channel subfamily V member 2 (TRPV2) functions as a stretch-sensitive calcium channel, with overexpression in the sarcolemma of skeletal and cardiac myocytes leading to detrimental calcium influx and triggering muscle degeneration. In our previous pilot study, we showed that tranilast, a TRPV2 inhibitor, reduced brain natriuretic peptide levels in two patients with muscular dystrophy and advanced heart failure. Building on this, we performed a single-arm, open-label, multicenter study herein to evaluate the safety and efficacy of tranilast in the treatment of advanced heart failure in patients with muscular dystrophy.
View Article and Find Full Text PDFDo the gait domains change in PD patients with freezing of gait during their 'interictal' period?
BMC Geriatr
January 2025
Department of Neurology, Xinhua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Objectives: Freezing of Gait (FOG) is one of the disabling symptoms in patients with Parkinson's Disease (PD). While it is difficult to early detect because of the sporadic occurrence of initial freezing events. Whether the characteristic of gait impairments in PD patients with FOG during the 'interictal' period is different from that in non-FOG patients is still unclear.
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