Supporting Orphaned Diseases in the Global Community
The Styrke Foundation is dedicated to bridging the critical gap between promising pre-clinical research and the development of life-changing therapies for children with rare diseases.
What is a Rare Disease?
A rare disease, also known as an orphan disease, is a medical condition that affects a relatively small number of people compared to the general population. The specific definition of “rare” can vary across countries, but it generally refers to conditions affecting fewer than 1 in 2,000 individuals.
The Global Impact of Rare Diseases
Despite their low prevalence, rare diseases collectively have a significant impact on public health worldwide. They can cause severe physical and mental disabilities, often leading to reduced quality of life and increased healthcare costs. Furthermore, many rare diseases are chronic and require lifelong medical care.
Challenges in Research and Funding
Rare diseases face numerous challenges in terms of research and funding. Here are some of the key reasons why they are often overlooked:
1. Small Patient Populations: The limited number of individuals affected by rare diseases makes it difficult to conduct large-scale clinical trials. This can hinder the development of effective treatments and diagnostic tools.
2. Lack of Profitability: Pharmaceutical companies may be reluctant to invest in research and development for rare diseases due to the small potential market size. This can lead to a lack of commercial interest and limited funding opportunities.
3. Diagnostic Challenges: Many rare diseases are difficult to diagnose, often leading to delayed or incorrect diagnoses. This can result in significant suffering for patients and their families, as well as missed opportunities for early intervention.
4. Limited Awareness: Rare diseases are often less visible than more common conditions, leading to a lack of public awareness and advocacy. This can make it difficult to secure funding and support for research initiatives.
Defining
Rare & Ultra-Rare Diseases
Rare Disease
Affecting less than 100,000 individuals worldwide
Ultra-Rare Disease
Affecting less than 50,000 individuals worldwide
A focus on Rare & Ultra Rare Disease
The Styrke Foundation’s screening process identifies programs that align with our mission and meet specific criteria for funding in these areas:
- A rare or ultra-rare disease
- Hematology focused
- Syndromes with consistent clinical presentation across variants caused by multiple gene variants
- Inherited, monogenic disorders
- Gene deficiency characterized by loss of function with no competing activity
- Vector delivery system suitability (to ensure manufacturing and toxicity): diseases/syndromes that are well suited for ex-vivo corrected CD34+ cells that can differentiate into erythroid progenitor cells which engraft into the bone marrow
- Manufacturing process consistency, to enable long-term decentralized manufacturing strategy
- Terminal, i.e. no autologous, curative solution
How Styrke is Tackling this Challenge
Our Five Tenets
How we tackle rare disease through funding. Every donation makes a difference to a child’s life.