Cargando…
Moving towards clinical trials for mitochondrial diseases
Primary mitochondrial diseases represent some of the most common and severe inherited metabolic disorders, affecting ~1 in 4,300 live births. The clinical and molecular diversity typified by mitochondrial diseases has contributed to the lack of licensed disease‐modifying therapies available. Managem...
Autores principales: | Pitceathly, Robert D.S., Keshavan, Nandaki, Rahman, Joyeeta, Rahman, Shamima |
---|---|
Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
John Wiley & Sons, Inc.
2020
|
Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8432143/ https://www.ncbi.nlm.nih.gov/pubmed/32618366 http://dx.doi.org/10.1002/jimd.12281 |
Ejemplares similares
-
Mitochondrial transcript maturation and its disorders
por: Van Haute, Lindsey, et al.
Publicado: (2015) -
Redox signalling and mitochondrial stress responses; lessons from inborn errors of metabolism
por: Olsen, Rikke K. J., et al.
Publicado: (2015) -
Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects
por: Baruteau, Julien, et al.
Publicado: (2017) -
Clinical presentation and outcome of riboflavin transporter deficiency: mini review after five years of experience
por: Jaeger, Bregje, et al.
Publicado: (2016) -
Opportunities and challenges for antisense oligonucleotide therapies
por: Kuijper, Elsa C., et al.
Publicado: (2020)