{"id":1436,"date":"2026-06-14T21:26:50","date_gmt":"2026-06-14T21:26:50","guid":{"rendered":"https:\/\/curetaok1.org\/?page_id=1436"},"modified":"2026-08-14T22:02:58","modified_gmt":"2026-08-14T22:02:58","slug":"research-timeline","status":"publish","type":"page","link":"https:\/\/curetaok1.org\/?page_id=1436","title":{"rendered":"Research timeline"},"content":{"rendered":"\t\t
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<\/p>\n

In brief<\/h2>\n

This page will eventually show the timeline of our research efforts \u2014 a roadmap from where we are today to a causal therapy for TAOK1-NDD. We are currently at the stage of exploring all the possibilities: analyzing the available directions, talking to scientists, and meeting every month to put the plan together step by step. We hope to be able to publish a concrete timeline here soon. Below, we explain where we stand \u2014 and give an honest answer to the question every family asks itself: “how long might this take?”<\/p>\n

Where are we today?<\/h3>\n

We are at the stage that, in the rare disease world, is called mapping the possibilities. Before we invest the first funds we raise, we want to understand the full picture: which therapeutic approaches being developed for other rare neurodevelopmental disorders could apply to TAOK1-NDD, which research teams around the world are working on the TAOK1 gene and related pathways, and which preparatory steps \u2014 above all, the patient registry and the natural history study \u2014 we need to complete so that any future therapy can be rigorously tested at all.<\/p>\n

Our working group meets every month. We continuously review the literature, consult with researchers, and learn from foundations that have walked this road before us. Once the plan is ready, we will publish it on this page \u2014 with stages, goals, and measures of progress, so that every donor and every family can follow where we stand.<\/p>\n

How long might the road to a treatment take? An honest answer<\/h3>\n

No one knows the exact answer \u2014 and anyone who gives you a specific date is simply guessing. What we can honestly do is draw on the experience of other genetic diseases to give expectations a realistic frame.<\/p>\n

The most frequently cited example is spinal muscular atrophy (SMA). The gene responsible for the disease, SMN1, was discovered in 1995. The following decade (roughly 2000\u20132010) was spent on preclinical research in animal models, the first human clinical trials began in 2011, and the first drug \u2014 nusinersen \u2014 was approved in the US in 2016. From the discovery of the gene to the first therapy, then, about 21 years passed. But here is what matters most: from the start of clinical trials to the approval of as many as three different therapies, less than ten years went by \u2014 once the scientific foundation was in place, the pace increased dramatically.<\/p>\n

Does TAOK1-NDD have to wait just as long? Not necessarily \u2014 and there are rational reasons for that. The TAOK1 gene was linked to the disease in 2019, in an era when therapeutic platforms for rare genetic diseases (gene therapies, antisense oligonucleotides, and others) already exist and are proven in practice \u2014 pioneers like the SMA community had to help create them from scratch. Knowledge about the gene itself is also growing exceptionally fast: only six years passed between the first description of the condition and the largest cohort study.<\/p>\n

Realistically, then, one has to think in terms of at least a decade \u2014 with the hope that ready-made therapeutic platforms, a well-prepared patient registry, and effective research funding will make it possible to shorten that road.<\/p>\n

The most important caveat<\/h3>\n

The figures above are only an estimate based on the history of other diseases \u2014 not a forecast, and certainly not a promise. Every disease is different: how quickly a therapy for TAOK1-NDD emerges depends on factors that no one can predict today \u2014 the results of basic research, which therapeutic approach turns out to be the right one, the availability of funding, and plain scientific luck, good and bad. It may go faster than the historical examples suggest; it may go slower. We promise one thing: we will communicate our progress transparently, and we will feed no one false hope or false pessimism.<\/p>\n

What brings us closer to the goal already today?<\/h3>\n

The paradox of rare diseases is that the pace of the road to a treatment is largely determined by things that can be done right away: a well-designed patient registry and natural history study (without them, no clinical trial can be planned), complete submission of variants to the ClinVar database, building relationships with research teams, and stable funding. That is exactly what we are focusing on now \u2014 and every family and every donor can play a real part in it. More information coming soon.<\/p>\n

Sources<\/h2>\n
    \n
  1. 25 years of the SMN genes: the Copernican revolution of spinal muscular atrophy.<\/em> 2021. https:\/\/pmc.ncbi.nlm.nih.gov\/articles\/PMC7783429\/<\/a><\/li>\n
  2. Spinal Muscular Atrophy: The Past, Present, and Future of Diagnosis and Treatment.<\/em> 2023. https:\/\/www.ncbi.nlm.nih.gov\/pmc\/articles\/PMC10418635\/<\/a><\/li>\n
  3. U.S. Food and Drug Administration (FDA). Rare Diseases: Natural History Studies for Drug Development. Draft Guidance for Industry.<\/em> 2019. https:\/\/www.fda.gov\/regulatory-information\/search-fda-guidance-documents\/rare-diseases-natural-history-studies-drug-development<\/a><\/li>\n
  4. Elkhateeb N. et al. Expanding the phenotype and genotype spectrum of TAOK1 neurodevelopmental disorder and delineating TAOK2 neurodevelopmental disorder.<\/em> Genetics in Medicine, 2025;27(3). https:\/\/www.gimjournal.org\/article\/S1098-3600(24)00282-X\/fulltext<\/a><\/li>\n<\/ol>\n

    \u00a0<\/p>\n

    This content is for informational purposes only and does not replace medical advice. Last updated: August 2026.<\/u><\/b><\/p>\n

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