Dravet syndrome pipeline review 2018 - Summary in Spanish for Dravet families.

Los avances en torno al tratamiento del síndrome de Dravet en los últimos años han seguido pasos agigantados. En los últimos 5 años hemos experimentado una explosión en el número de programas en desarrollo para tratar la enfermedad: de tener solo Diacomit y nada más a tener Diacomit y 14 más en desarrollo.​​​​​​​ Este texto es un resumen en Español del Dravet syndrome pipeline review 2018 dirigido a familias, con el texto no solo traducido sino también ajustado a los intereses de aquellos que tienen un hijo/a con síndrome de Dravet y su entorno.

After May 2018 patients will have the right to request access to their data files. They will have the right to withdraw their data from a database that is not being used in the way that the patient through it would operate. And they will have the right to obtain these files generated by a particular data holder, for example a gene testing lab, and get the usable file and share it with as many studies and registries as the patient wants. The EU General Data Protection Regulation gives patients the Right to Access, the Right to be Forgotten, and the Data Portability right.

There is no doubt that AAVs will support the development of many gene therapies for genetic diseases, but for many diseases AAVs are too small to carry a copy of the gene that patients need. There is a strong need to find non-AAV alternatives that can provide a suitable gene therapy option for those diseases caused by mutations in large genes. Within Dravet syndrome, these next-generation therapies are being led not by companies but by academic groups with the support of patient organizations. This is a review of these programs and how they are attempting to develop a gene therapy for treating Dravet syndrome.

we expect in 2018 the first approval of a treatment for Dravet syndrome in the US, the results of clinical trials with three new therapeutics (ZX008, ataluren and OV935/TAK935), and the initiation of the first clinical trial ever done with a disease-modifying therapy designed to treat Dravet syndrome (OPK88001). 2018 is going to be a good year.