- Drug development for ultra|rare diseases🔍
- Rare Disease Clinical Trial Networks; Request for Information and ...🔍
- Canada's regulatory approach to drugs for rare diseases🔍
- Small Data Challenges of Studying Rare Diseases🔍
- Rare diseases🔍
- HORIZON|JU|IHI|2023|04|04|two|stage🔍
- Innovations in Therapy Development for Rare Diseases Through the ...🔍
- What You Need to Know About the Rare Disease Cures Accelerator ...🔍
Rare Disease Cures Accelerator|Data
Drug development for ultra-rare diseases: What happens when N=1?
By treating ASOs for new ultra-rare diseases as repurposed drugs, the FDA can reduce the amount of toxicology and preclinical studies needed to ...
Rare Disease Clinical Trial Networks; Request for Information and ...
In 2018, for the first time ever, a majority of new molecular entities approved by the FDA were orphan drugs to treat rare diseases. However, of ...
Canada's regulatory approach to drugs for rare diseases: orphan ...
Advice on clinical trial applications, clinical trial design in small populations and planning New Drug Submissions · Accelerated Review Pathways.
Small Data Challenges of Studying Rare Diseases - JAMA Network
Support for rare disease research continues today. In 2016, the US Food and Drug Administration awarded $23 million over 4 years to support ...
By changing medical practice, pursuing first or best-in-class medicines, focusing on cutting edge research and development and utilising new digital health ...
HORIZON-JU-IHI-2023-04-04-two-stage - Funding & tenders
Deliver novel rare/ultra-rare disease-specific methodological approaches2 to transform the way treatments are developed with a view towards ...
Innovations in Therapy Development for Rare Diseases Through the ...
The Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP) is an FDA-funded effort to overcome these persistent challenges. By ...
What You Need to Know About the Rare Disease Cures Accelerator ...
The National Organization for Rare Disorders (NORD), Critical Path Institute (C-Path), and the US Food and Drug Administration (FDA) hosted ...
FDA Diversity Requirement: Impact on Rare Disease Drug ... - Avalere
Increasing clinical trial diversity presents unique challenges for rare disease treatments due to population size, disease heterogeneity, and low awareness.
Sarepta Therapeutics | Biopharmaceutical Company for Rare ...
Sarepta is engineering solutions for rare diseases with science that is on the forefront of precision genetic medicine. Our platforms include: gene therapy, RNA ...
Exploring biomarker strategies in rare disease therapeutics
Innovative biomarker strategies and regulatory flexibility from the FDA are helping to streamline rare disease therapy development, addressing ...
Data silos are undermining drug development and failing rare ...
The current research and development landscape sets too many projects up for unnecessary failure, particularly in the rare disease sphere, and ...
ROI and Rare Disease: Retooling the 'Gene' Value Machine
Framework proposes three strategies designed to address the unique challenges of personalized and genetic therapies for rare diseases—and ...
How logistics shape the success of rare disease trials
At two upcoming events on rare disease clinical trials and data ... rare disease treatments, and how alternatives to placebo arms should be ...
RDCA-DAP - Value of Integrated Data and Analytics in ... - YouTube
Value of Integrated Data and Analytics in Rare Disease Drug Development C-Path's Rare Disease Cures Accelerator–Data and Analytics Platform ...
Alexion is a global biopharmaceutical company focused on developing life-changing therapies for people living with rare disorders.
Clinical Trial Shows Positive Results for Potential Treatment to ...
An international, phase 3 clinical trial led by investigators at Mass General Brigham could improve the treatment of a rare disease that can ...
Therapeutic Orphan No More: Role for Clinical Pharmacology and ...
However, for some rare diseases even when multiple drugs have been approved, unmet medical need can still persist. ... In this issue, Konieczny ...
Clinical trial shows positive results for potential treatment to combat ...
An international, phase 3 clinical trial led by investigators at Mass General Brigham could improve the treatment of a rare disease that can ...
Innovative research methods for studying treatments for rare diseases
We identified 16 research strategies for studying rare disease. Innovative clinical trial methods minimize sample size requirements (n=4) and ...