Bridging the Gap Between Patients and Scientists:

Hope for Luka Inc. serves as a crucial link between patients with muscular dystrophy and the scientific community dedicated to finding solution

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  • Dr. Annemieke Aartsma-Rus

    Parents Becoming Research Coordinators

    By fostering connections between families and leading researchers, Hope for Luka Inc. catalyzes groundbreaking collaborations and accelerates the pace of discovery. Moreover, the organization plays a pivotal role in fundraising efforts, empowering parents to initiate independent pre-clinical trials and studies tailored to their children's unique needs.

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  • An "Open Effort" Mindset

    The "open effort" research funding model emphasizes on collaboration and transparency in the fight against Duchenne Muscular Dystrophy (DMD). By making research findings accessible to pharmaceutical and research organizations, this model fosters a collaborative environment where multiple entities can work together to refine and enhance the treatment approach.

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Research Pipelines

Research Pipeline: ∆17-44 lncRNA Study

The Use of lncRNA to Preserve and Increase the Production of Dystrophin With ∆17-44 Exons Deletion

The research aims to study the use of lncRNA to preserve and increase the production of dystrophin and to create a mice model of the genetic deletion/duplication to test the research's findings in an in-vivo setting.

Dr. Aartsma-Rus (Leiden University Medical Center in the Netherlands See their website here) is currently confirming the use of lncRNA in another study published in the Q4 of 2024, to preserve dystrophin protein for Luka's deletion. This study will benefit exon-skipping studies, rare brain disease research as well as cancer research. 

Dr. Aartsma-Rus of Leiden University Medical Center in the Netherlands is considered to be one of the best translational geneticist researcher in Muscular Dystrophy worldwide.

Cost of study

Leiden University Medical Center in the Netherlands has funded preclinical discovery costs of the study.

Start Date March , 01st 2025 Started
Targeted Completion Date March, 01st 2026
Number of Patients Benefitting  All In-Frame Deletions
∆17-44  Pre-clinical Costs $86,000 Funded by LUMC

     

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          Our Values

          Hope for Luka is an organization deeply rooted in the principle of compassion as well as consciousness, recognizing them as the cornerstones of our mission. We are dedicated to advancing independent pre-clinical trials, and our approach is driven by a heartfelt commitment to children with rare diseases.

          See Mazi Keyghobadi speaking on our values: 

          Our team is composed of researchers, scientists, and members who view patients not just as subjects of study, but as individuals whose lives hold invaluable potential. This compassionate perspective fuels our pursuit of innovation and excellence, ensuring that every step we take is motivated by empathy and a genuine desire to make a difference.

          By fostering an environment where compassion thrives, we believe we can achieve groundbreaking progress and bring hope to those who need it most.