Development of new effective therapies for rare diseases

Published by European Commission (EC)

GrantRare Disease

This topic aims at supporting activities that are enabling or contributing to one or several expected impacts of destination 3 “Tackling diseases and reducing disease burden”. To that end, proposals under this topic should aim for delivering results that are directed, tailored towards and contributing to some of the following expected outcomes:

Researchers and developers make the best use of the state-of-the-art knowledge and resources for a fast and effective development of new therapies for rare diseases.
Researchers and developers increase the development success rate of therapies for rare diseases by employing robust preclinical models, methods, technologies, validated biomarkers, reliable patient reported outcomes and/or innovative clinical trials designs.
Developers and regulators move faster towards market approval of new therapies for rare diseases (with currently no approved treatment option) due to an increased number of interventions successfully tested in late stages of clinical development.
Healthcare professionals and people living with a rare disease get access to new therapeutic interventions and/or orphan medicinal products.

View this resource Bookmark this resource

Research Grant for Rett Syndrome

Published by International Rett Syndrome Foundation

GrantRett Syndrome

IRSF believes that research into a variety of Rett and Rett-related topics is necessary to create treatments and eventual cures for every individual living with Rett syndrome. We proudly invest in such research at every stage: from basic science that can change the way we think about MeCP2 to clinical research that directly impacts potential treatments. We also help connect Rett researchers with external funding opportunities.

View this resource Bookmark this resource

Chan Zuckerberg Initiative

Published by Chan Zuckerberg Initiative

Chan Zuckerberg Initiative

In addition to traditional grantmaking to support people and organizations working toward our missions in Science, Education, and within our communities, the Chan Zuckerberg Initiative makes venture investments in impact-focused companies, builds tools and products that we can scale and give away for free, and supports capacity building to achieve progress across our work.

View this resource Bookmark this resource

Research Grants for Angelman Syndrome

Published by Angelman Syndrome Foundation

Angelman SyndromeGrant

The ASF Scientific Advisory Committee identified the following areas of unmet need in AS research. Priority will be given to the following topics:

Projects studying or correcting the heterozygous effect of non-UBE3A genes in deletion.
Projects studying the potential results of increasing UBE3A after therapies or for some subtypes of AS.
Projects studying delivery of therapies and potential for improvement.
Symptomatic therapies that impact the daily life of people with Angelman syndrome and their families.
Proposals that do not address these topics are still welcome and will be given full consideration.

View this resource Bookmark this resource