Results 71 to 80 of about 2,204 (179)

The Illness Narratives of Children and Young People With Spinal Muscular Atrophy: A Scoping Review

Journal of Advanced Nursing, Volume 82, Issue 9, Page 8375-8390, September 2026.
ABSTRACT Aim(s) This review seeks to explore the illness narratives of children and young people focusing on their healthcare trajectories; the right to health; and the kind of stories told about them. Design This scoping review adopts a narrative approach to analyse how the illness experience of Spinal Muscular Atrophy is represented in the literature,
Marcela González‐Agüero, Constanza Quezada, Valentina Turén, Josefa Camelio, Martina De Filippi, Caroline Bradbury‐Jones, Julie Taylor   +6 more
wiley   +1 more source

Anion‐Exchange Membrane Chromatography for the Separation of Empty and Full Adeno‐Associated Viral Capsids

Biotechnology Journal, Volume 21, Issue 8, August 2026.
Graphical Abstract and Lay Summary The anion‐exchange membrane elution chromatography for the separation of empty and full AAVs is explored and optimized. The role played by important parameters like flowrate, gradient slope, membrane loading, and feed residence time is studied in detail.
Luca Ossi, Samuele Delfino, Helena Marie, Michael Schulte, Mattia Sponchioni   +4 more
wiley   +1 more source

Fatal thrombotic microangiopathy case following adeno-associated viral SMN gene therapy

, 2022
Adeno-associated virus (AAV) gene therapies are highly promising, such as the onasemnogene abeparvovec (Zolgensma) in spinal muscle atrophy (SMA).
Guillou, Julia, Péréon, Yann, Allain-Launay, Emma, Barnérias, Christine, Debord, Camille, Denis, Manon, Mercier, Sandra, de Pellegars, Alice, Frémeaux-Bacchi, Véronique, Roussey, Gwenaëlle, Porcheret, Florence, Desguerre, Isabelle   +11 more
core   +1 more source

Improved therapeutic approach for spinal muscular atrophy via ubiquitination‐resistant survival motor neuron variant

Journal of Cachexia, Sarcopenia and Muscle
Background Zolgensma is a gene‐replacement therapy that has led to a promising treatment for spinal muscular atrophy (SMA). However, clinical trials of Zolgensma have raised two major concerns: insufficient therapeutic effects and adverse events.
Joonwoo Rhee, Jong‐Seol Kang, Young‐Woo Jo, Kyusang Yoo, Ye Lynne Kim, Sang‐Hyeon Hann, Yea‐Eun Kim, Hyun Kim, Ji‐Hoon Kim, Young‐Yun Kong   +9 more
doaj   +1 more source

New Therapeutics Options for Pediatric Neuromuscular Disorders

Frontiers in Pediatrics, 2020
Neuromuscular disorders (NMDs) of Childhood onset are a genetically heterogeneous group of diseases affecting the anterior horn cell, the peripheral nerve, the neuromuscular junction, or the muscle.
Marina Flotats-Bastardas, Andreas Hahn
doaj   +1 more source

Targeted Interventions in HAM/TSP: Emerging Therapies and Future Directions – A Narrative Review

Health Science Reports, Volume 9, Issue 8, August 2026.
ABSTRACT Background and Aim Human T‐Lymphotropic virus type 1 (HTLV‐1) infection can lead to HTLV‐1‐associated myelopathy/tropical spastic paraparesis (HAM/TSP), a debilitating chronic neurological disease characterized by progressive lower limb spasticity and autonomic dysfunction.
Meygol Mirzaei Rezaei, Mahdi Khosravi Nia, Kasra Allaei Rouzbahani, Maryam Kazemi, Negar Asghari Hosori, Mehdi Norouzi, Farzaneh Sotoudegan, Narges Eslami, Negar Ariamand, Vahid Shahnavaz, Arash Letafati, Sayed‑Hamidreza Mozhgani   +11 more
wiley   +1 more source

SMA: REGISTRIES, BIOMARKERS & OUTCOME MEASURES: P.187 Systematic literature review of the economic burden and economic evaluations in spinal muscular atrophy [PDF]

, 2020
peer reviewedNew treatments in spinal muscular atrophy (SMA) have led to a complete change in the pattern in the use of health care resources in this disease.
Hiligsmann, Mickaël, Servais, Laurent, Dangouloff, Tamara   +2 more
core   +1 more source

AAV-aMTD-Parkin, a therapeutic gene delivery cargo, enhances motor and cognitive functions in Parkinson's and Alzheimer’s diseases

Pharmacological Research
Neurodegenerative disorders, such as Parkinson's disease (PD) and Alzheimer's disease (AD), have a global prevalence and profoundly impact both motor and cognitive functions. Although adeno-associated virus (AAV)-based gene therapy has shown promise, its
Seokwon Lee, Mingu Kang, Seungwoo Lee, Sangsun Yoon, Yeonjin Cho, Dongjae Min, Daye Ann, Jisoo Shin, Young-Ki Paik, Daewoong Jo   +9 more
doaj   +1 more source

From Nexavar to Zolgensma: Has Indian Compulsory Licensing Law Evolved?

The compulsory licence issued in India in 2012 for Bayer's cancer drug Nexavar remains the seminal instance of section 84 of the Patents Act, 1970 in action. The case is frequently read in India either as proof that patient health trumps patent rights or
Ujjawal Solanki
core   +1 more source

Εξελίξεις της γονιδιακής θεραπείας στη νωτιαία μυϊκή ατροφία [PDF]

, 2021
Η Νωτιαία Μυϊκή Ατροφία (Spinal Muscular Atrophy) είναι μία νευρομυϊκή διαταραχή που κληρονομείται με αυτοσωμικό υπολειπόμενο τρόπο. Χαρακτηρίζεται από τον εκφυλισμό των πρόσθιων κεράτων των άλφα κινητικών νευρώνων του νωτιαίου μυελού με αποτέλεσμα τη ...
Σαλαγιάννη Χρυσάνθη-Αγγελική, Salagianni Chrysanthi-Angeliki   +1 more
core