September 13, 2020

Gene Therapy, Dravet syndrome, Clinical trials

Gene therapy for Dravet syndrome – 2020 update

September 13, 2020

Gene Therapy, Dravet syndrome, Clinical trials

Gene therapy for Dravet syndrome – 2020 update

There are multiple gene therapy and oligonucleotide programs in development for Dravet syndrome including those that supply and extra copy of the SCN1A gene and those that boost expression from the healthy SCN1A gene copy. Clinical trials have already started, with Stoke Therapeutics initiating the first clinical trial with a disease-targeting therapy in Dravet syndrome in summer 2020. Behind Stoke, gene therapies are approaching the clinic with Encoded Therapeutics having the most advanced clinical candidate and preparing for trials in 2021.

Dracaena Consulting

August 30, 2018

Dravet syndrome, Epilepsy, Orphan drugs, Rare diseases

Main lessons from the European Congress on Epilepsy 2018

Dracaena Consulting

August 30, 2018

Dravet syndrome, Epilepsy, Orphan drugs, Rare diseases

Main lessons from the European Congress on Epilepsy 2018

Every other year the International League Against Epilepsy organises a major epilepsy medical congress in Europe called the European Congress on Epilepsy (ECE). This year I attended the main three days of the ECE addition in Vienna, looking at the field partly as a drug developer and partly as a patient advocate working on behalf of rare epilepsy patient communities. Here is the list of what I found the most interesting at the ECE 2018 meeting.

Dracaena Consulting

July 23, 2018

Dravet syndrome, Drug Discovery

DRAVET SYNDROME DRUG DEVELOPMENT PIPELINE REVIEW 2018

Dracaena Consulting

July 23, 2018

Dravet syndrome, Drug Discovery

DRAVET SYNDROME DRUG DEVELOPMENT PIPELINE REVIEW 2018

The 2018 Dravet Syndrome Pipeline and Opportunities Review provides a review and analysis of 14 drug candidates in development for the treatment of Dravet syndrome, including 9 products that have received orphan drug designations. It also includes an analysis of the competitive landscape and evaluates current and future opportunities of the Dravet syndrome market.

Dracaena Consulting

September 25, 2017

Dravet syndrome, Drug Discovery, Epilepsy, Orphan drugs, Personalized medicine, Rare diseases

Genes vs syndromes at the International Epilepsy Congress in Barcelona

Dracaena Consulting

September 25, 2017

Dravet syndrome, Drug Discovery, Epilepsy, Orphan drugs, Personalized medicine, Rare diseases

Genes vs syndromes at the International Epilepsy Congress in Barcelona

Should we talk about syndromes based on the gene that causes them or should we talk about them (and treat them) based on the clinical characteristics that they display? Earlier this month, the epilepsy community gathered in Barcelona for the 32nd International Epilepsy Congress and there was a debate between genetic and symptom-base syndrome classification. This debate goes beyond semantics, and has important regulatory and access implications.

Dracaena Consulting

June 23, 2017

Orphan drugs, Epilepsy, Drug Discovery, Dravet syndrome

Dravet Syndrome Drug Development Pipeline Review 2017

Dracaena Consulting

June 23, 2017

Orphan drugs, Epilepsy, Drug Discovery, Dravet syndrome

Dravet Syndrome Drug Development Pipeline Review 2017

June 23 is a special day for families of people with Dravet syndrome. It is the International Dravet Syndrome Awareness Day, that in 2017 celebrates its 4th edition. That's why today we announce the publication of the 2017 Dravet Syndrome Pipeline and Opportunities Review, a market research publication that provides an overview of the global therapeutic landscape of Dravet syndrome.

Dracaena Consulting

October 28, 2016

Drug Discovery, Dravet syndrome, Orphan drugs, Rare diseases, Epilepsy, Impatient patients

Impatient patients

Dracaena Consulting

October 28, 2016

Drug Discovery, Dravet syndrome, Orphan drugs, Rare diseases, Epilepsy, Impatient patients

One day I sent an e-mail that changed my life. During my training as a scientist, I had learned how to study what goes wrong with the brain and to research how we could fix it. Then one day I sent an e-mail to a patient.