Research Pipelines
Current Research Study in Pipeline
N=2 Double Exons-Skipping Research - See Details
| Start Date | April, 1st 2025 |
| Targeted Completion Date | March, 31st 2027 |
| Number of Patients Exons 6 & 7 Skipping May Benefit | 300 Patients |
| Total Costs | $4,000,000 |
Next Research Study in Pipeline
∆7-44 Deletion lncRNA Study - See Details
| Start Date | March , 01st 2025 |
| Targeted Completion Date | March, 01st 2026 |
| Number of Patients Benefitting | All In-Frame Deletions |
| Total Costs - (Preclinical Discovery) | Self-funded by LUMC |
How We Prioritize Our Research Study Funding
Each patient or parent of patients participating in our organization represent a population of patients that share the same mutation.
We prioritize research studies based on the severity of the mutations in our pipeline. This means more severe mutations have smaller window of opportunity to find any sort of solutions compared to less severe.