Emma

4 years Switzerland AGO2

Diagnosed patient number 22 worldwide!

In the summer of 2020 our 5-month-old daughter suddenly jerked her arms and legs repeatedly and cried uncontrollably. We watched in horror, quickly googled the symptoms, and rushed to the emergency room.  After repeatedly being asked if Emma had lost skills, an overnight surveillance and brain activity measurement, the report was:

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Emma was started that day on very high doses of two anti-epileptics to combat her brain-damaging seizures. We worried one of the drugs would impair her vision and ability to drive, but the doctors said this should be the least of our worries. We left the hospital after three days with a sense of despair and very little information on how to cope.

In the following weeks, Emma brutally suffered from side effects and cried day and night. We were both on part-time leave from work and mobilized every resource we could for round-the-clock support: grandparents, aunt, a currently unemployed friend, and emergency child care services at our own cost. We organized night shifts so we could sleep a few hours at a time. With our hearts bleeding and zombie-like fatigue, our anxiety boiled over in everyday situations and we fought a lot.  We were at a loss of how to function in such extreme circumstances? How to deal with a prognosis that destroys your imagined future? How to live with anxiety about the future? How did other families support their child and partner? We longed for support from others who had gone through a similar experience to lean on as a couple and to learn from.

To uncover the cause of the epilepsy, Emma underwent a brain scan, blood analysis and gene test. We hoped they wouldn’t find anything but they did: a de novo mutation in the gene AGO2 that wasn't seen in either of us. Emma was diagnosed patient number 22 worldwide. The updated report was:

The 21 known patients had both physical and mental impairments, and reading through their case reports broke our hearts. The clinicians knew little more. Just as we eased off one of the drugs and Emma’s laughter came back, this diagnosis made our world crumble down. The lack of options felt like our hearts had been torn out, stabbed with a dagger and thrown down a cliff only to be run over by a truck. “Determine to feel well” felt like a cruel joke rather than a strategy to cope.

Neither the doctors nor Google knew much about this novel rare disease and it wasn’t clear what information to trust.

How did other children develop? Did their condition get worse over time? What therapeutic strategies did other parents try to help their children learn to walk and speak? Were there other children who developed further than the known patients? We wished a reliable source of information would exist.

Because Christoph and me both have a background in research, we couldn't just sit this prognosis out and we started searching for options. We soon found out that there was hope: Thanks to tremendous scientific progressgene therapies have finally come to patients and rare disease patient organizations are acting as successful incubators (e.g. FAST for Angelman snydrome, Progeria Research Foundation and CureSMA). Patient organizations can play a critical role to cross the gap between government-funded basic research and private sector-funded clinical trials that some have called the “valley of death”. Patient organizations must advance the search for treatments to a stage where the development of a profitable drug looks sufficiently likely for pharma, biotech and venture funds to become engaged. This involves amongst others the following three tasks, each of which is explained in more detail in our research roadmap

To enable our daughter and others like her to develop like other children, we decided to found a patient organization dedicated to improving the quality of life of those affected by AGO2 syndrome. Our three-pronged mission brings to life what we learned through our own painful experience: 

Emma is back on a good developmental path but a return of epilepsy would put a halt to this. Other affected children neither learnt to walk nor talk. We need to find treatments now so she keeps laughing, learns to speak and can attend regular school. And so we can save other affected children like her.

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