Zolgensma Therapy

Helping with

ZOLGENSMA® genetherapy

At Bethesda Children’s Hospital of the Reformed Church in Hungary we have been delivering chargeable gene replacement therapy to treat spinal muscular atrophy (corrected) at the official request of parents since October 2019.

Up to today, the hospital has broadened its experience by effectively treating over 15 patients.

Below we have summarised for you all the most important details to ensure that, as parents, you are fully informed about the entire treatment process.

ZOLGENSMA® is a genetherapy used in the treatment of children diagnosed with SMA.

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The child has to be younger than 30months and less than 13,5kg at the therapy

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Please make sure that you read carefully the medical conditions your child has to meet.

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A 3-month period of treatment takes place once the medicine has been administered

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Total cost of the entire genetherapy treatment: 2.132.000 EUR

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Please read carefully all the main points of the therapy procedures

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What is Zolgensma and to whom it can be applied?

ZOLGENSMA® (onasemnogene abeparvovecxioi) is a genetherapy used in the treatment of children diagnosed with muscular dystrophy (spinal muscular atrophy (SMA), which was granted valid marketing authorisation as a registered drug treatment across the entire territory of the European Union on 18 May 2020. (In the U.S. prescription, the use of Zolgensma genetherapy is possible only in children under 2 years of age.)

Based on clinical experience to date, an international recommendation has been issued on the suitability of the medicine, according to which genetherapy is recommended for children who do not exceed the weight limit of 13.5 kg.

The Bethesda hospital is continuously studying the relevant international medical literature and we have to emphasize that the results of clinical trials are based on data from infants and asymptomatic infants and children who does not exceed the weight limit of 13.5 kg and there are limited data on efficacy and safety in the advanced stage of the disease. Based on the facts written before, there are very little available international experience and data in terms of the risk and benefit ratio of genetherapy in children over 2 years of age and children over 13.5 kg. Considering this fact the Hospital has taken the decision that until we have a sufficient number of literature recommendations, we will not be able to perform genetherapy for children over 30 months (2,5 years) of age or for children who are heavier than 13.5 kg.

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SUMMARIZING: The last date for administering Zolgensma is the 30th month from the birth of the child.

We have to emphasize that the final decision about the genetherapy treatment can be made only if all the relevant medical documents of the child have been reviewed by the Hospital and the medical team takes its decision always individually in each case.

The Hospital can not be held responsible for the efficacy, side effects and complications of Zolgensma therapy, as well as the costs incurred.

 

The primary conditions for admission to the programme are the following ZOLGENSMA® genetherapy indications:

Either
5q spinal muscular atrophy (SMA) with biallelic SMN1 gene mutation and the underlying clinical diagnosis is SMA-1
Or
5q SMA with biallelic SMN-1 gene mutation with at most 3 copies of the SMN2 gene

Additional medical criteria to be met in order for the treatment to be carried out:

Your child:

  1. does not require invasive ventilation
  2. has no acute or uncontrolled infection
  3. does not take medication for neuromuscular disorders (exception for Nusinersen) or diabetes
  4. has not undergone immunosuppressive therapy (steroids, cytostatica, immunoglobulin) within the 3 months prior to the proposed date of undergoing ZOLGENSMA® genetherapy
  5. in the event your child was born prematurely, their present age is not less than the full-term pregnancy
  6. is younger than 30 months (2,5 years) at the date of the ZOLGENSMA® genetherapy
  7. is not heavier than 13,5 kg

Please make sure your child fits all the above criteria before you apply for Zolgensma Therapy. You also have to be able to prove the above with documents received from your local treating doctor.

Apply here
For your information we are listing below the reasons that may preclude your child undergoing ZOLGENSMA® gene therapy:
  • diagnosis of spinal muscular atrophy not confirmed by genetic testing
  • positive serologies such as HIV, Hepatitis-A or Hepatitis-C
  • invasive respiratory support
  • anti-AAV9 titer result above 1:50 ratio (ELISA immunoassay)
  • clinically significant elevation of hepatic enzymes (GGT > three times the upper limit of normal value), serum ammonia levels above the normal range
  • elevated bilirubin (≥3.0 mg/dL),
  • elevated creatinine (≥ 1.8 mg/dL),
  • haemoglobin levels below 8 g/dL or over 18 g/dL,
  • white blood cell count over 20 G/L
  • abnormally high serum troponin-I levels
  • abnormal renal function values
  • abnormal coagulation parameters, known coagulation disorder
  • abnormally high or low platelet count
  • signs of acute infection (bronchitis, fever, vomiting, diarrhoea, etc.)
  • medication for musculoskeletal or nervous system disorders (except Nusinersen) or diabetes,
  • immunosuppressive therapy (steroid, cytostatic treatment, immunoglobulin) during the previous three months
  • any infection occurred within one month before the scheduled date of administration
  • active vaccination within one month of the scheduled date of administration

Genetherapy treatment includes the following health services, supplementary and related services:

A 3-month period of treatment takes place once the medicine has been administered, which will require you moving to Budapest, Hungary. During this 3-month period we are following the child physical status; carrying out different control examinations, laboratory tests, dietary examination, consultation with specialists etc.)

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Gene therapy treatment – the health care fees payable for the treatment – includes the following health services, and supplementary and related services:

  • full examination of the patient
  • procuring, preparing and administering the medicine
  • hospitalisation for the time the medicine is administered (max. 4-5 days)
  • 3 months’ treatment delivered as outpatient care:
    • following the child physical status continously;
    • carrying out different control examinations regulary,
    • regulary laboratorytests,
    • dietary examination,
    • consultations with specialists etc
    • calibrating medication doses, delivering supplementary immunosuppressive drug therapy
    • conducting additional examinations depending on the child’s actual condition (imaging examinations, consultation with specialists etc.)
  • regular medically prescribed gymnastic therapy (depend on the physical status of the patient) after the first month after treatment
  • dealing with complications, if necessary through hospitalisation (intensive care if necessary)
  • calibrating medical devices in the event of non-invasive ventilation
    • repairing or replacing medical devices in the event of failure,in the case of a machine provided by a hospital
  • providing dietary advice, optimising nutrition, tube feeding if necessary and regular tube replacement.
  • providing culturally appropriate meals during period of hospitalization (for 3 persons)
  • providing a case manager to help the patient’s family in the hospital everytime they have to come for
  • examinations, meetings in person etc.

Costs involved in delivering the entire genetherapy treatment

Total cost of the entire genetherapy treatment: 2.132.000 EUR

Costs does NOT included in the treatment:

  • travel to and from Hungary, accommodation and living costs during your stay in Hungary
  • patient care beyond the specified 3-month treatment period (in your country)
  • the price of the medical technology equipment and devices recommended by the medical specialists of the Hospital in the course of the gene therapy treatment for use subsequent to the completion of said 3-month gene therapy treatment and which are customized to the child (you have the opportunity to buy these equipment in our Hospital, but it is not obligatory).
  • other medical care that is not related to the gene therapy treatmet

Do you need help with all the above costs?

EliMo Foundation is here to help you with Fundraising, covering the costs of treatment and everything extra you need for traveling and accommodation. We are here to help with all the arrangements as well.
Optional services may include: help with finding accommodation, interpreter services, car rental or airport transfer.

Apply here for help

The main steps of the application and authorization procedure of the Zolgensma therapy

1.Before you Apply! Please first read carefully all the information about the Zolgensma Therapy and please make sure that your child’s condition meets all the medical criteria above in order to receive the therapy.

Please note: You can Apply for the Fundraising Help with EliMo Foundation at any time of the process. The page also contains all the extra services the Foundation can help you with along the therapy.

2. If you meet the medical conditions above, then you can fill out the Application form for the Zolgensma therapy. Please make sure that you also upload the mandatory documents/files listed on the application page!

>> Application Form for Zolgensma Therapy <<

3. After receiving the form and the requested documents the medical team may ask — based on the supplied data of the child—for further documents (that means, further examination of the child’s suitability continues or not).

4. Based on the information provided in the Application form our medical team takes a decision whether to accept the application.

5. If the child’s application has been accepted the Hospital will send the a Price Offer for performing the gene therapy (The price offer does not mean any obligation regarding the gene therapy treatment carried out by the Hospital).

6. In case of accepting the application you take the decision as parents to have your child undergo gene therapy at Bethesda Children’s Hospital in Budapest, Hungary.

7. You have to provide documented proof that sufficient funds are available to cover the entire cost of the treatment OR if you need help with fundraising for the costs of the treatment, please Apply for Fundraising help here.

8. After you accepted the price offer and you have the available funds the Hospital sends a Medical record and information list about all the medical records and information our medical team needs (only English version is accepted).

9. The hospital also contacts your child’s local treating doctor, requesting additional medical documentation if necessary.

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Zolgensma_treatment

10. Once our doctors have studied your child’s complete medical records, Bethesda Children’s Hospital will make a final decision on your child’s suitability for gene therapy,  provided there are no contraindications on  undertaking your child’s treatment.

11. Bethesda Children’s Hospital plans the gene therapy and draws up a treatment schedule.

12. Local treating doctor signs an official statement (we send a form to fill and sign) to the effect that they will undertake aftercare of your child at the local SMA Centre in your country of residence upon your return home.

13. You will need to move to Budapest, Hungary 2 weeks prior to the proposed date of the gene therapy being administered. It is important you have to bring all equipment your child uses (devices related to ventilation and every medical equipment for movement etc.)

Please note: if you need help with travel plans, visa or accommodation, please contact the Foundation and read the information about the services the Foundation can provide here.

14. Upon your arrival we meet you at Bethesda Children’s Hospital for an examination of your child and for a detailed briefing and to answer any further questions you may have, to sign the necessary legal documents. Because of the examinations you have to ensure your child doesn’t eat or drink before the meeting for at least 2 hours.

15. The total cost of the entire gene therapy treatment is transferred to Bethesda Children’s Hospital’s bank account.

16. Bethesda Children’s Hospital orders the medicine and arranges for the medicine to be imported (the medicine will reach the Hospital within 10 days of the date of ordering)

17. Medicine is administered in the Bethesda Hospital, hospitalisation may take up to 4-5 days.

18. Your child will be under the treatment of our doctors for 3 months aftercare therapy as outpatient care in Budapest (Hungary).

Please note: if you need help arrangement of accommodation and extra services for this 3 months period please contact the Foundation here.

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