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Treatment for Spinal Muscular Atrophy (SMA)

Combination of medication and stem cell therapy offers the best possible in SMA treatment.

In recent years, several innovative therapies for the treatment of spinal muscular atrophy (SMA) have been approved in many countries. 

Spinraza (nusinersen) was the first medication approved for the treatment of SMA. It received approval in December 2016. This marked a significant milestone as it became the first-ever FDA-approved treatment specifically developed for SMA.

Prior to the approval of Spinraza, there were no targeted treatments available for SMA. Spinraza’s approval represented a breakthrough in the field of SMA research and offered hope to individuals and families affected by this rare genetic neuromuscular disorder. Since then, other treatments have also been approved, broadening the treatment options for SMA patients.

In addtion to Spinraza, Zolgensma (onasemnogene abeparvovec) was approved by the FDA in May 2019 and Evrysdi (risdiplam) was approved in August 2020 for the treatment of SMA, and followed by many other countries.

Spinraza (Nusinersen)

Spinraza is the first medicine approved for the treatment of SMA that increases survival motor neuron (SMN) protein levels. It is administered through spinal fluid injections and helps improve motor function and muscle strength in SMA patients.

Zolgensma (Onasemnogene Abeparvovec)

Zolgensma is a gene therapy, it involves a one-time intravenous infusion of a modified viral vector containing a functional copy of the survival motor neuron 1 (SMN1) gene. This therapy aims to address the underlying cause of SMA by providing a functional SMN1 gene to help produce the SMN protein.

Evrysdi (Risdiplam)

Evrysdi (risdiplam) is an oral medication, it is a small molecule splicing modifier designed to increase the production of functional SMN protein from the SMN2 gene. It can be taken orally, making it more convenient than other treatment options that require invasive procedures.

These newer treatment options provide additional choices for individuals and families affected by SMA. Each treatment has its own benefits, considerations, and administration methods. The decision regarding the most suitable treatment should be made in consultation with healthcare providers who specialize in managing SMA, taking into account individual needs, priorities, and medical circumstances.

Effective yet expensive

It is often the case that medical treatments or therapies that are effective are also expensive.  These new treatments for SMA can be expensive. The cost of these therapies can present significant challenges for individuals and families seeking access to them.

While the high cost of SMA treatments can present barriers to access, efforts are being made to address these challenges. Some potential solutions include:

  1. Negotiations and Discounts: Insurance companies or government health agencies may negotiate with pharmaceutical companies to lower the treatment’s cost or secure discounts, making it more affordable for patients.

  2. Patient Assistance Programs: Pharmaceutical companies often provide patient assistance programs, such as copay assistance or free drug programs, to support individuals who cannot afford the cost of treatment.

  3. Government Support: In some cases, governments may provide subsidies or financial assistance programs to help patients access expensive treatments.

  4. Research and Development: Continued research and development in the field of SMA may lead to the discovery of alternative treatments that are more cost-effective.

It is important for individuals and families affected by SMA to work closely with healthcare providers, patient advocacy groups, and insurance providers to explore all available options for accessing and affording these treatments.

Each country has its own healthcare policies and regulations regarding the reimbursement and coverage of medications like Spinraza.

In some countries, Spinraza may be fully or partially covered by public healthcare programs or private insurance, while in others, patients may need to pay out-of-pocket or seek alternative funding sources. The reimbursement policies for Spinraza can depend on various factors including the country’s healthcare system, regulatory policies, and budgetary constraints.

Affordable solution is available now!

Learn about our treatment package for SMA with Spinraza and Evrysdi. Contact us and get a quote.

Spinraza has been approved in China for the treatment of SMA since 2019.

Evrysdi has been approved in China for the treatment of SMA patients aged above 2 months old, and in June 2023 it received approval for the treatment of SMA patients aged 16-day old, this approval was based on the latest studies of Evrysdi.

Zolgensma is currently under a phase III clinical trial in China.

Steps to access the treatment

1. Learn about Spinraza and Evrysdi.

2. Contact us and get information about our package.

3. Submit your medical reports for our doctors to make acceptance.

4. Once accepted, you can start to prepare the travel visa and be ready for the journey towards healthier life.

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