As an antisense oligonucleotide (ASO) therapy, nusinersen sodium injection targets the core cause of SMA with targeted efficacy:
1. Targeted Repair of Key Genes: It specifically acts on the SMN2 gene, regulating its splicing process to promote the production of more functional SMN protein—a "necessity" for the survival and normal function of motor neurons.
2. Efficient Delivery to Lesions: Administered via lumbar puncture, the drug is directly injected into the cerebrospinal fluid (CSF), ensuring it accurately reaches SMA-affected areas in the spinal cord. This minimizes drug loss during delivery and maximizes therapeutic effects.
3. Protecting the Foundation of Motor Function: By increasing SMN protein levels, the drug reduces motor neuron damage, fundamentally slowing or even stopping muscle atrophy and laying the groundwork for preserving or restoring patients' mobility.
Validated by international multicenter clinical trials and long-term follow-ups, nusinersen sodium injection demonstrates stable and significant efficacy across different SMA types and age groups:
1. Type 1 SMA (Predominantly Infants): Increases the 2-year survival rate from a historical 50% to over 80%; helps patients master key motor skills such as independent sitting and voluntary head control; reduces reliance on ventilators and lowers the risk of severe complications.
2. Type 2 & 3 SMA (Children to Adults): Effectively slows disease progression, preventing further decline in limb mobility and respiratory function; some patients maintain their current motor abilities or even achieve mild improvements; early intervention in pre-symptomatic patients (e.g., newborns) supports near-normal motor development.
3. Long-Term Efficacy Guaranteed: Five-year follow-up data show that motor function improvements in treated patients remain stable without significant regression. Since its global approval in 2016, the therapy has benefited over 100,000 patients in more than 60 countries, covering all SMA subtypes.
Additionally, authoritative clinical trials (e.g., EMBRACE, ENDEAR, CHERISH) provide robust evidence: Among 274 enrolled patients, the motor milestone achievement rate was 51% higher than the control group (p<0.00001), and HINE-2 motor function score improvements were 26% higher than the control group (p<0.0003). Studies on adolescent and adult patients also confirm that their HFMSE (Hammersmith Functional Motor Scale Expanded) and RULM (Revised Upper Limb Module) scores continue to improve 26 months after treatment.
Leveraging national drug price negotiation policies and the hospital's public welfare mission, Beijing JingduChildren's Hospital offers nusinersen sodium injection at a price far lower than international markets, making this "life-saving drug" accessible to more patients:
Treatment Setting | Cost per Injection (USD) | Total Cost for 4 Loading Doses (USD) | First-Year Cost (USD) | Included Services |
Beijing JingduChildren's Hospital | ~7,600 | ~30,400 | ~30,400 (Initial Phase) | Drug cost, injection fees, ward care, pre-treatment tests, cold-chain storage |
United States (No Subsidy) | 125,000 | 500,000 | 750,000 (6 Injections Total) | Only base drug cost (excludes tests, care, and other additional fees) |
Australia (No Subsidy) | 87,500 | 350,000 | 525,000 (6 Injections Total) | Only base drug cost (some tests require additional payment) |
Comparatively, the total cost of the initial 4 loading doses at Beijing Jingdu Children's Hospital saves over 720,000 USD compared to the U.S. and over 500,000 USD compared to Australia. This reduces the financial burden on families by more than 90%, allowing more patients to receive standardized treatment with peace of mind.
Recognizing the unique needs of SMA patients (especially children), Beijing JingduChildren's Hospital has developed a "pre-treatment, in-treatment, post-treatment" care system to deliver professionalism and compassion:
Pre-Treatment: Meticulous Preparation to Ease Anxieties
1. Multidisciplinary consultation: Neurologists, rehabilitation specialists, and pediatricians jointly assess patients to develop personalized treatment plans based on age, SMA subtype, and physical condition.
2. Streamlined medical processes: Assists with health insurance policy consultations, hospitalization appointments, and test scheduling to avoid unnecessary travel for families.
3. Science popularization and psychological support: Explains drug mechanisms, treatment cycles, and precautions to families, alleviating their anxiety.
In-Treatment: Professional Escort for Safety
1. Experienced medical team: Skilled healthcare providers perform lumbar puncture injections, monitoring vital signs throughout to minimize risks.
2. Scientific treatment scheduling: Strictly follows the timeline of "Day 1, Day 14, Day 28" for the first 3 injections; patients are observed for 6 hours after each injection to ensure no adverse reactions.
3. Comfortable environment: Child-friendly wards are equipped with toys and picture books to reduce children’s fear of treatment; nutritional dietary advice is provided to maintain patients' physical condition.
Post-Treatment: Sustained Follow-Up for Worry-Free Recovery
1. Convenient maintenance treatment: The 4th dose is provided with professional cold-chain packaging, allowing injection at local medical institutions (if approved by doctors); regular reminders for rechecks and maintenance doses are sent every 4 months.
2. Rehabilitation guidance and follow-up: Rehabilitation specialists develop home-based training plans and conduct combined online/offline follow-ups to adjust plans as needed.
3. Long-term health management: Establishes personalized health records for patients, documenting treatment effects and recheck data to track disease progression dynamically.
If your family member (especially a child) is struggling with SMA and seeking scientific, affordable, and compassionate treatment, the nusinersen sodium injection program at Beijing JingduChildren's Hospital is a reliable choice，Jingdu is one of the few institutions in China qualified to receive international SMA patients. Here, you'll find solid efficacy, affordable pricing, and caring service—all working together to protect patients' mobility and help them regain confidence in life.
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