Results 161 to 170 of about 36,350 (272)

Novel drugs approved by the EMA, the FDA and the MHRA in 2024: A year in review

British Journal of Pharmacology, Volume 182, Issue 7, Page 1416-1445, April 2025.
Abstract In the past year, the European Medicines Agency (EMA), the Food and Drug Administration (FDA) and the Medicines and Healthcare Products Regulatory Agency (MHRA) authorised 53 novel drugs. While the 2024 harvest is not as rich as in 2023, when 70 new chemical entities were approved, the number of ‘orphan’ drug authorisations in 2024 (21) is ...
Stavros Topouzis, Andreas Papapetropoulos, Steve P. H. Alexander, Miriam Cortese‐Krott, Dave A. Kendall, Kirill Martemyanov, Claudio Mauro, Nithyanandan Nagercoil, Reynold A. Panettieri Jr, Hemal H. Patel, Rainer Schulz, Barbara Stefanska, Gary J. Stephens, Mauro M. Teixeira, Nathalie Vergnolle, Xin Wang, Péter Ferdinandy   +16 more
wiley   +1 more source

Eteplirsen in the treatment of Duchenne muscular dystrophy

Drug Design, Development and Therapy, 2017
Kenji Rowel Q Lim,1 Rika Maruyama,1 Toshifumi Yokota1,2 1Department of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, 2The Friends of Garrett Cumming Research & Muscular Dystrophy Canada, HM Toupin Neurological Science ...
Lim KRQ, Maruyama R, Yokota T
doaj  

No sex difference in mutations rates of Duchenne muscular dystrophy. [PDF]

, 1980
N. Yasuda, K Kondo
openalex   +1 more source

Leber Hereditary Optic Neuropathy: Support, Genetic Prediction and Accurate Genetic Counselling Enhance Family Planning Choices

Clinical &Experimental Ophthalmology, Volume 53, Issue 3, Page 292-301, April 2025.
ABSTRACT With the increased availability of genetic testing and the addition of mitochondrial genetic variants on disease panels, accurate genetic counselling for individuals and families affected by, or at risk of, Leber hereditary optic neuropathy (LHON) is becoming increasingly relevant.
Lisa S. Kearns, Sandra E. Staffieri, David A. Mackey   +2 more
wiley   +1 more source

The population genetics of Duchenne: natural and artificial selection in Duchenne muscular dystrophy. [PDF]

, 1986
James Edwards
openalex   +1 more source

Prime Editing: A Revolutionary Technology for Precise Treatment of Genetic Disorders

Cell Proliferation, Volume 58, Issue 4, April 2025.
This review outlines the latest research advancements in prime editing technology, delivery strategies, and the challenges that must be addressed to fully realize its therapeutic potential, emphasizing the high potential of prime editing in the remission or cure of genetic diseases. ABSTRACT Genetic diseases have long posed significant challenges, with
Mengyao Li, Yi Lin, Qiang Cheng, Tuo Wei
wiley   +1 more source

Longitudinal trajectories of muscle impairments in growing boys with Duchenne muscular dystrophy. [PDF]

PLoS One
Vandekerckhove I, Van den Hauwe M, Dewit T, Molenberghs G, Goemans N, De Waele L, Van Campenhout A, De Groote F, Desloovere K.   +8 more
europepmc   +1 more source

Duchenne Muscular Dystrophy - Family in a Crisis

, 1970
Mohammad Robed Amin, Chowdhury Chironjib Borua, Kaji Shafiqul Alam, Fazle Rabbi Chowdhury, Rabiul Jahan Sarkar, Md Abul Kashem Khandaker, MA Faiz   +6 more
openalex   +2 more sources

Identifying Concepts of Physical Activity Which Are Clinically Meaningful to Patients and Care Providers: A Systematic Review of Qualitative Research

Clinical and Translational Science, Volume 18, Issue 4, April 2025.
ABSTRACT Physical activity (PA) is indispensable for overall health. Sub‐optimal PA is linked to reduced quality of life (QOL) and premature death. In clinical research and therapeutics development, defining aspects of PA that are meaningful to patients and care providers is essential for designing tailored interventions, identifying individual ...
Candice Taguibao, Salma Ajraoui, Jake Centra, Kieran F. Reid, Christina Daskalopoulou, Alberto Conde Freniche, Alan L. Hamilton, Astrid M. H. Horstman, Benjamin X. Collins, Jessilyn Dunn, Elena S. Izmailova   +10 more
wiley   +1 more source

Deep characterization of females with heterozygous Duchenne muscular dystrophy mutations. [PDF]

J Neurol
Riguzzi P, Sabbatini D, Fusto A, Vianello S, Merlo B, Zangaro V, Capece G, Gorgoglione D, Sorarù G, Bariani R, Calore C, Bauce B, Martini M, Mutterle A, Bello L, Pegoraro E.   +15 more
europepmc   +1 more source