Results 111 to 120 of about 6,842,005 (300)

Onasemnogene Abeparvovec in Patients With SMA: Interim Results of the RESTORE Registry in Japan

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective There are limited real‐world data regarding the safety and effectiveness of onasemnogene abeparvovec (OA; Zolgensma) infusion, a one‐time gene replacement therapy, for Japanese patients with spinal muscular atrophy (SMA). We aimed to improve understanding of the real‐world outcomes for OA in Japan.
Kayoko Saito, Kamal Benguerba, Ken Tsuchida, Kazushige Yazawa, Isao Tsumiyama, Hiromitsu Kayama, Sandra P. Reyna, Farid Khan, Richard S. Finkel   +8 more
wiley   +1 more source

"Acute Respiratory Disease" [PDF]

Medical Clinics of North America, 1947
C H, RAMMELKAMP, J H, DINGLE
openaire   +4 more sources

Global, regional, and national burden of chronic obstructive pulmonary disease and its attributable risk factors from 1990 to 2021: an analysis for the Global Burden of Disease Study 2021

Respiratory Research
Background Chronic obstructive pulmonary disease (COPD) continues to be a significant issue, leading to premature death or reduced quality of life. It’s important to assess the current burden of COPD and its risk factors on a geographical basis to guide ...
Zhufeng Wang, Junfeng Lin, Lina Liang, Feifei Huang, Xiaoyin Yao, Kang Peng, Yi Gao, Jinping Zheng   +7 more
doaj   +1 more source

Respiratory Disease [PDF]

Epidemiologic Reviews, 2000
openaire   +2 more sources

Cognitive and Neuroimaging Divergence Between Juvenile and Adult FUS Amyotrophic Lateral Sclerosis

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disorder characterized by progressive motor neuron degeneration. Fused in sarcoma (FUS)‐associated juvenile ALS (jALS) represents a distinct and aggressive subgroup with rapid deterioration and poor prognosis.
Alexandra V. Jürs, Marcel Naumann, Annaliis Lehto, Hanna Schön, Jens Kurth, Johannes Prudlo, Andreas Hermann, Elisabeth Kasper   +7 more
wiley   +1 more source

Antibody dynamics for heterologous boosters with aerosolized Ad5-nCoV following inactivated COVID-19 vaccines

Human Vaccines & Immunotherapeutics
The COVID-19 pandemic has underscored vaccination as a crucial strategy for reducing disease severity and preventing hospitalizations. Heterologous boosters using aerosolized Ad5-nCoV following two doses of inactivated vaccine have demonstrated superior ...
Canjie Chen, Tian Tang, Zhao Chen, Lan Chen, Jinling Cheng, Fang Li, Jing Sun, Jingxian Zhao, Yanqun Wang, Qihong Yan, Jincun Zhao, Airu Zhu   +11 more
doaj   +1 more source

Integration of Serum Neurofilament Light Chain and Cortical Dysfunction Improves Diagnostic Accuracy in ALS

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective To determine whether integration of serum neurofilament light chain (NfL) and cortical dysfunction improves diagnostic accuracy in amyotrophic lateral sclerosis (ALS) when applied alongside the Gold Coast criteria (GCC). Methods In this prospective study, 148 participants with suspected ALS were recruited (101 ALS and 47 with ALS ...
Aicee Dawn Calma, Nathan Pavey, Yukiko Tsuji, Amirul Ab Ghapar, Mehdi A.J. van den Bos, Bronwen Orden, Grace Lee, Linda Mekhael, Julie Ryder, Cláudia Santos Silva, David McDonald, Fakhria Kakar, Con Yiannikas, Matthew C. Kiernan, David A. Brown, Parvathi Menon, Steve Vucic   +16 more
wiley   +1 more source

Re‐Purposing Sapropterin (Kuvan) for ACTA2‐Related Multisystemic Smooth Muscle Dysfunction Syndrome: A Translational Mechanistic and First‐In‐Human Therapeutic Report

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Multisystemic smooth muscle dysfunction syndrome (MSMDS) is an ultra‐rare, ACTA2‐related disorder characterized by severe cerebrovascular disease, aortic aneurysms, and smooth muscle dysfunction. Using molecular dynamics simulations and in silico drug screening, we identified that sapropterin dihydrochloride (Kuvan) is a candidate capable of ...
Moran Hausman‐Kedem, Noa Bielopolski, Vijai Krishnan, Shane Wald‐Altman, Shelly I Shrian, Omer Bar‐Yosef, Livia Kapusta, Claire Shamber, Patricia L. Musolino, Miguel Weil   +9 more
wiley   +1 more source

Technical evaluation of Turbuhaler® use in children with bronchial asthma: combination of a checklist and inhalation parameters

BMC Pulmonary Medicine
Background The Turbuhaler® is a dry powder inhaler (DPI) that is commonly used in the treatment of asthmatic patients aged 5 years and above. Nevertheless, the technique of using the Turbuhaler® in the real world remains ambiguous.
Lina Liang, Ruogang Huang, Yun Li, Zhufeng Wang, Kang Peng, Junfeng Lin, Feifei Huang, Xiaoyin Yao, Jinping Zheng, Yi Gao   +9 more
doaj   +1 more source

Respiratory diseases [PDF]

Postgraduate Medical Journal, 1992
openaire   +2 more sources