Paving the way for Amelia’s childhood

Flawless delivery of gene therapy for young spinal muscular patient

When Amelia* from Chile was diagnosed with spinal muscular atrophy (SMA), she needed a one-time gene replacement therapy to save her life. World Courier worked around the clock to support in delivering the product on time.

The situation

At just six months old, Amelia was diagnosed with SMA, a rare genetic disease that results in the loss of motor neurons and progressive muscle wasting. The condition is found in just 1 in 10,000 children born around the world and, if left untreated, more than 90% of patients may die or require permanent ventilation by the age of two¹.

“SMA affects a child’s ability to crawl, sit up and control their own head movements, even breathe, or swallow. There is no guaranteed cure, but treatment and support from a young age can reduce symptoms and improve quality of life.”

— Amelia’s doctor

Faced with this news, Amelia’s family set out to obtain a one-time, potentially life-saving gene replacement therapy produced in the U.S.

Amelia, like many other vulnerable patients living in remote areas, struggle to access cutting-edge gene therapies. Many products require storage at a very specific temperature range, as low as -70°C and only have a shelf life of 48 hours. Failure to meet these strict requirements can lead to the degradation of the therapy, have devastating ramifications for patients’ health and wellbeing, and cost millions of dollars to rectify.

The World Courier team made sure that the dose reached Amelia on time and in good condition.

The solution

The World Courier team harnessed our unique global network of local logistics and regulatory experts to make certain that the dose reached Amelia on time and in good condition.

The box used for transportation was a 30L global dry ice (GDI) thermal container, chosen for its ability to keep its contents at a consistent temperature of -70°C, whatever the external conditions.

To minimize the impact of routing disruptions due to the pandemic and safeguard against delays, we fitted a GPS tracker in addition to our existing advanced tracking capabilities. This allowed us to monitor the location of the thermal container from departure through to where Amelia was being treated.

At every stage, our experts reviewed the documents, permits and custom requirements. With our unique experience and insight, we made sure that each piece of documentation was compliant with local regulations in the US and Chile to prevent any delays in the customs process.

“We understand the impact of what we do, particularly in sensitive cases like this one. It is why we went the extra mile by arranging a police escort from the airport to the hospital. The dedication and commitment of our team combined with the assistance of the police escort ensured our timely arrival and guaranteed that nothing would hinder our mission to deliver life-saving medicine to a child in need.”

— Tatiana Marante, Regional Vice President, Latin America

The outcome

Amelia received her gene replacement therapy safely and on time, thanks to our well-established global logistics infrastructure and team of dedicated people with their local regulatory, custom and transport expertise.

At World Courier we understand the importance of on-time and in-temperature delivery for every shipment. We are committed to supporting all of our clients with their life-saving medications so they can reach the people who need them most, wherever they are in the world.

*To protect patient privacy, all identifying details have been changed. Visual portrayals of patients are stock images.

¹ Finkel RS, et al. Neurology. 2014;83(9):810-817.

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