Be a Hope for Asrin Efe's SMA Type 1 Treatment
Donation protected
(Not: Türkçe metin, "Gofundme aracılığıyla nasıl ödeme yapılır?" ve banka hesap detayları aşağıdadır)
Please help to save Asrin Efe's life!
I am starting a crowdfunding campaign for Asrin Efe Gulkan (born on 2nd Nov 2020) who is the son of my sister and brother-in-law Serife Naz and Serkan Gulkan who are still in early stages of their marriage.
Asrin Efe was diagnosed with Spinal Muscular Atrophy (SMA) type 1 – a life limiting neuromuscular disease that life expectancy of the majority of children is less than 18 months (please see the references below for details.
However, Asrin Efe has a chance due to being one of the smallest babies with SMA in the world if he can receive a one-time dose of a gene therapy called Zolgensma. As SMA is a rare disease, Novartis charges a huge fee to cover their R&D costs and make a profit: Zolgensma costs $2.4 million – with additional expenses such as travel, accommodation and consultancy fees, the total amount of reaches ~$2.5 million (currently ~£1.94m). For Novartis to approve Zolgensma therapy, a child must not reach 2 years or 13kg – as this negatively affects the effectiveness and the safety of the treatment. Asrin Efe is currently 2 months old (as of October 2021), thus we have a fight against the clock while his illness affects his ability to breathe, swallow, control head movements and crawl each passing day .
We believe that there are many people willing to help Asrin Efe. For this purpose, please donate as much as you can – even if it’s a fiver – and give Asrin Efe and his family the chance to give life a shot. Please also share with anyone you feel would be pleased to help.
Thank you!
Below, you can find further information on Zolgensma, Asrin Efe, and his parents.
For updates and to reach the father or mother directly:
Email: [email redacted]
Instagram: sma_asrinefe
Facebook: Sma Asrın Efe
Twitter: smaasrinefe
Youtube: https://youtu.be/pNVAx2UVfQ8
Extra information for UK donors: The NHS offers a drug called Spinraza to SMA patients, which is an effective therapy in the short-term, however it is invasive and only a partial solution and the costs to the taxpayer builds up continuously (especially for type 1 SMA patients). For a new therapy like Zolgensma to be offered by the NHS, it is thoroughly reviewed by the NICE committee (https://www.nice.org.uk/). The meeting for Zolgensma is expected to be held in October 2020 and the report is expected to be published in March 2021. The implementation of the therapy is likely to be much later than that, which is going to be too late for Asrin Efe. There are great developments in the field of rare disease genetics and prices are expected to drop significantly in 5-10 years’ time but not soon enough for Asrin Efe. (Note: the newly FDA approved Risdiplam is also not available via the NHS and will need to go through NICE similar to Zolgensma)
Further information:
1- NHS page on SMA: https://www.nhs.uk/conditions/spinal-muscular-atrophy-sma/types/
2- Zolgensma general information: https://smauk.org.uk/zolgensma
3- Blog post on the long-term value of Zolgensma by an SMA patient: https://www.statnews.com/2019/05/31/spinal-muscular-atrophy-zolgensma-price-critics/
Note: If you do not want to pay a tip to gofundme.com, then do not forget to change the drop box (see below photo) to "Other" and then the amount to "0". Gofundme.com will take a small fee when I send the final donation amount to the family so no need to add extra unless you personally want to
SMA Tip 1 Hastası Asrın Efe'nin desteğinize ihtiyacı var!
Baldızım Şerife Naz ve eşi Serkan Gükan'ın oğulları Asrın Efe Gülkan için bir bağıi kampanyası başlatmak istiyorum. Asrın Efe, maalesef ölümcül bir hastalık olan Spinal Müsküler Atrofi (SMA) Tip 1 teşhisi aldı. Asrın Efe'nin yaşayabilmesi için sadece 2 yaş (ve 13 kilo) altındaki bebekler için onaylanan ve $2.2 milyon bedelindeki Zolgensma isimli gen terapisini alması gerekiyor. Ulaşım, konaklama ve konsultasyon gibi diğer maliyetlerle birlikte toplanması gereken bağış miktarı ~$2.5 milyonu buluyor ve bu miktar yalnızca sizlerin desteğiyle başarılabilir. Az ya da çok yapacağınız her miktarda bağış Asrın Efe'nin sağlığına kavuşması için çok önemli.
Lütfen kampanyamızı tüm tanıdıklarınızla paylaşın.
Yardımlarınız için şimdiden teşekkür ederiz.
Aileye ulaşmak için iletişim adresleri aşağıdadır:
Websitesi: http://sma-asrinefe.com/
Youtube: https://youtu.be/pNVAx2UVfQ8
Instagram: sma_asrinefe
Twitter: smaasrinefe
Facebook: Sma Asrın Efe
Email: [email redacted]
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Please help to save Asrin Efe's life!
I am starting a crowdfunding campaign for Asrin Efe Gulkan (born on 2nd Nov 2020) who is the son of my sister and brother-in-law Serife Naz and Serkan Gulkan who are still in early stages of their marriage.
Asrin Efe was diagnosed with Spinal Muscular Atrophy (SMA) type 1 – a life limiting neuromuscular disease that life expectancy of the majority of children is less than 18 months (please see the references below for details.
However, Asrin Efe has a chance due to being one of the smallest babies with SMA in the world if he can receive a one-time dose of a gene therapy called Zolgensma. As SMA is a rare disease, Novartis charges a huge fee to cover their R&D costs and make a profit: Zolgensma costs $2.4 million – with additional expenses such as travel, accommodation and consultancy fees, the total amount of reaches ~$2.5 million (currently ~£1.94m). For Novartis to approve Zolgensma therapy, a child must not reach 2 years or 13kg – as this negatively affects the effectiveness and the safety of the treatment. Asrin Efe is currently 2 months old (as of October 2021), thus we have a fight against the clock while his illness affects his ability to breathe, swallow, control head movements and crawl each passing day .
We believe that there are many people willing to help Asrin Efe. For this purpose, please donate as much as you can – even if it’s a fiver – and give Asrin Efe and his family the chance to give life a shot. Please also share with anyone you feel would be pleased to help.
Thank you!
Below, you can find further information on Zolgensma, Asrin Efe, and his parents.
For updates and to reach the father or mother directly:
Email: [email redacted]
Instagram: sma_asrinefe
Facebook: Sma Asrın Efe
Twitter: smaasrinefe
Youtube: https://youtu.be/pNVAx2UVfQ8
Extra information for UK donors: The NHS offers a drug called Spinraza to SMA patients, which is an effective therapy in the short-term, however it is invasive and only a partial solution and the costs to the taxpayer builds up continuously (especially for type 1 SMA patients). For a new therapy like Zolgensma to be offered by the NHS, it is thoroughly reviewed by the NICE committee (https://www.nice.org.uk/). The meeting for Zolgensma is expected to be held in October 2020 and the report is expected to be published in March 2021. The implementation of the therapy is likely to be much later than that, which is going to be too late for Asrin Efe. There are great developments in the field of rare disease genetics and prices are expected to drop significantly in 5-10 years’ time but not soon enough for Asrin Efe. (Note: the newly FDA approved Risdiplam is also not available via the NHS and will need to go through NICE similar to Zolgensma)
Further information:
1- NHS page on SMA: https://www.nhs.uk/conditions/spinal-muscular-atrophy-sma/types/
2- Zolgensma general information: https://smauk.org.uk/zolgensma
3- Blog post on the long-term value of Zolgensma by an SMA patient: https://www.statnews.com/2019/05/31/spinal-muscular-atrophy-zolgensma-price-critics/
Note: If you do not want to pay a tip to gofundme.com, then do not forget to change the drop box (see below photo) to "Other" and then the amount to "0". Gofundme.com will take a small fee when I send the final donation amount to the family so no need to add extra unless you personally want to
SMA Tip 1 Hastası Asrın Efe'nin desteğinize ihtiyacı var!
Baldızım Şerife Naz ve eşi Serkan Gükan'ın oğulları Asrın Efe Gülkan için bir bağıi kampanyası başlatmak istiyorum. Asrın Efe, maalesef ölümcül bir hastalık olan Spinal Müsküler Atrofi (SMA) Tip 1 teşhisi aldı. Asrın Efe'nin yaşayabilmesi için sadece 2 yaş (ve 13 kilo) altındaki bebekler için onaylanan ve $2.2 milyon bedelindeki Zolgensma isimli gen terapisini alması gerekiyor. Ulaşım, konaklama ve konsultasyon gibi diğer maliyetlerle birlikte toplanması gereken bağış miktarı ~$2.5 milyonu buluyor ve bu miktar yalnızca sizlerin desteğiyle başarılabilir. Az ya da çok yapacağınız her miktarda bağış Asrın Efe'nin sağlığına kavuşması için çok önemli.
Lütfen kampanyamızı tüm tanıdıklarınızla paylaşın.
Yardımlarınız için şimdiden teşekkür ederiz.
Aileye ulaşmak için iletişim adresleri aşağıdadır:
Websitesi: http://sma-asrinefe.com/
Youtube: https://youtu.be/pNVAx2UVfQ8
Instagram: sma_asrinefe
Twitter: smaasrinefe
Facebook: Sma Asrın Efe
Email: [email redacted]
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Yusuf Sakin
Organizer
England
