Author：Medical Editor Jason
Content:
What is SMA, and what is its incidence rate?
Spinal Muscular Atrophy (SMA) is a genetic disorder resulting from mutations in the SMN1 gene. It primarily impacts motor neurons in the spinal cord, causing muscle weakness and atrophy. Globally, approximately one in every 10,000 newborns is affected by SMA, with Type I, the most severe form, commonly manifesting in infancy.
How is SMA treated?
SMA can be treated through various methods, including:
Gene therapy: One such option is Zolgensma, a one-time injection that addresses the root cause by repairing the mutated SMN1 gene.
Disease-modifying therapies: These include Nusinersen (Spinraza), an injectable drug, and Risdiplam, an oral medication. Both work by increasing SMN protein levels to alleviate symptoms.
Adjuvant therapies: These encompass physical therapy, respiratory support, and nutritional management, all aimed at enhancing the patient's quality of life.
Is there a cure for SMA patients?
Currently, the primary objectives of SMA treatment are to delay disease progression and alleviate symptoms. Gene therapy and disease-modifying therapies have made significant strides in improving patient survival rates and quality of life. However, achieving a complete cure still requires additional research and development.
Advantages and recent developments in SMA treatment in the United States
Advantages of SMA Treatment in the United States:
Innovative Medications: Innovative medications such as Zolgensma have been developed, which, despite their high cost, are considered highly effective gene therapy options.
Multidisciplinary Teams: Multidisciplinary teams specializing in SMA care are available in American hospitals. These teams comprise experts from various fields, including neurology, genetics, and physical therapy, ensuring a comprehensive approach to patient management.
Advanced Equipment: Advanced diagnostic and monitoring equipment, such as high-resolution MRIs, is used to assess and track changes in muscle function, aiding in the early detection of disease progression.
In terms of recent treatment advancements:
The FDA has approved Voyager, marking it as the first oral SMA treatment suitable for patients of all ages.
Promising new gene editing technologies, such as CRISPR, are currently undergoing clinical trials. These technologies aim to provide a more definitive repair of the mutated SMN1 gene, potentially leading to more effective treatment outcomes.
In summary, the United States offers cutting-edge technology, specialized multidisciplinary teams, and a range of treatment options for SMA, providing patients with enhanced treatment opportunities and improved prognosis.
Top hospitals and experts specializing in SMA treatment in the U.S
The United States is home to numerous hospitals and experts who are pioneers in the field of SMA diagnosis and treatment. Below are brief introductions to some of these prominent institutions and leading specialists:
Boston Children's Hospital: This institution has vast experience in early diagnosis and intervention for SMA, enabling timely and effective management of the disease.
Mayo Clinic: Offering a holistic approach to SMA treatment, Mayo Clinic incorporates gene therapy and rehabilitation support, ensuring comprehensive care for patients.
Texas Children's Hospital: As one of the leading trial centers for SMA gene therapy, this hospital is at the forefront of research and development in this field.
Top Experts:
Dr. John W. Day: Serving as the Director of the Neuromuscular Division and Clinic at Stanford University, Dr. Day is a renowned expert in SMA research, contributing significantly to advancements in the field.
Dr. Basil Darras: Dr. Darras holds the position of Director of the Neuromuscular Center and SMA Program, as well as Chief of the Clinical Neurology Department at Boston Children's Hospital. His expertise and dedication have made significant impacts on the care and management of SMA patients.
Collectively, these hospitals and experts leverage their extensive experience and cutting-edge technology to provide comprehensive and personalized medical services to SMA patients, significantly enhancing their quality of life and treatment outcomes.
The cost of SMA treatment in the United States
Gene Therapy (e.g., Zolgensma): Approximately $2,100,000 (one-time treatment).
Spinraza: $125,000 per dose (4 doses in the first year, followed by 3 doses annually thereafter).
Evrysdi: $300,000−400,000 per year.
Costs vary based on the treatment modality and individual patient needs.
For international patients, the cost of treating SMA without a U.S.-based insurance plan can be substantially higher than for U.S. residents, as they must pay full price. These medical expenses are often beyond the financial means of average families.
It is worth noting that some specialized U.S.-based cross-border medical service agencies can assist international patients in applying for cost reductions from hospitals. For example, Medebound Health once helped an Asian ACL orthopedic patient obtain a nearly 50% discount on the cost of a Bear surgery at the Hospital for Special Surgery (HSS) in the United States.
Challenges faced by international SMA patients
For families confronted with a diagnosis of a serious illness, accessing advanced therapies in the United States frequently appears unattainable due to several challenges.
Exorbitant cost of treatment: particularly gene therapy, it often discourages patients from pursuing such options.
Lack of localized solutions: Limited domestic treatment options and asymmetric patient information make it difficult for patients to make informed decisions.
Obstacles to cross-border treatment: Cross-border treatment presents obstacles such as language barriers, cultural differences, and difficulties in follow-up care.
So, is there a viable solution to help alleviate the burden on these patients?
The answer is yes, indeed there are ways to assist.
Cases: Asian SMA patients consulted with U.S. experts via remote video
Everyone experiences unique moments of joy throughout their life, and for Mr. Wang, one such moment was becoming a father for the first time.
As he cradled his newborn son in his arms, Mr. Wang's face lit up with joy, envisioning the future life of their trio. He felt that there couldn't be a more beautiful moment in the world.
However, his world was soon shattered by the diagnosis of Spinal Muscular Atrophy (SMA), a term that began to dominate his life.
Despite the initial sweetness of becoming a father, Mr. Wang was dealt a bitter blow.
In such a challenging situation, the patient and his family turned to MEDEBOUND HEALTH, a cross-border medical service agency with nine years of experience in overseas medical services and a remarkable 95% patient satisfaction rate.
Medebound HEALTH facilitated a telemedicine consultation for Mr. Wang's 1-year-old son, enlisting the expertise of Dr. Steven M. Wolf and Associate Director Patty McGoldrick from Mount Sinai Hospital in the United States. After thoroughly reviewing the child's medical history and condition, the two specialists offered the following comprehensive treatment recommendations:
Pharmacological Treatment:
The recommended treatment is Nusinersen (brand name: Spinraza), an FDA-approved drug that, through lumbar puncture injection into the cerebrospinal fluid, enhances the concentration of Survival Motor Neuron (SMN) protein.
Clinical studies have demonstrated significant improvements in motor function following treatment.
Adjuvant Therapies:
Nutritional Management: Close monitoring of feeding is advised, with consideration for gastrostomy if deemed necessary.
Respiratory Support: Regular assessment of respiratory function is crucial, as deterioration may necessitate non-invasive respiratory support or tracheotomy.
Scoliosis Management: Surgical intervention will be evaluated based on the progression of spinal curvature.
Physical and Occupational Therapy: Continuous rehabilitation training is essential to mitigate further muscle deterioration.
Prognosis and Follow-up Care:
Ongoing lumbar intrathecal injections are scheduled every four months, accompanied by continuous monitoring of the child's nutritional status, respiratory function, and skeletal health.
Emphasis is placed on the significance of early intervention to achieve optimal developmental milestones.
Medical Treatment in the United States
Following the video consultation, Mr. Wang's resolve to seek SMA treatment in the United States was solidified. In close partnership with Medebound HEALTH, Mr. Wang successfully navigated the following preparatory steps:
Hospital Selection: After conducting a thorough comparison of prices and services, Medebound HEALTH recommended the University of California, Los Angeles Medical Center (UCLA Medical Center), citing its reasonable costs and comprehensive medical services as key advantages.
Visa and Living Arrangements: Medebound HEALTH extended comprehensive support to Mr. Wang, encompassing visa processing, accommodation arrangements in the United States, airport pickup services, and daily life translation assistance, ensuring a seamless transition into his new environment.
Treatment Decision: Upon consulting with experts at the hospital, Mr. Wang opted for the Nusinersen drug treatment regimen, declining participation in clinical trials in favor of a more established therapeutic approach.
Treatment Process and Outcomes
At UCLA, the child has successfully undergone the first three injections of treatment and is now preparing for the fourth. Based on feedback provided by Mr. Wang, it is evident that the child's muscle function has undergone noticeable improvement following the treatment, resulting in an enhanced quality of life for the entire family. This positive outcome underscores the effectiveness of the medical intervention and the supportive care provided by the center.
Patient's Testimonial
Mr. Wang expressed his gratitude towards Medebound HEALTH for not only facilitating connections with top-tier hospitals and specialists but also for providing comprehensive and attentive full-process services. These services streamlined the otherwise complex process of seeking medical treatment overseas.
Specifically, the professional medical translation and escort services greatly mitigated language and cultural obstacles, enabling Mr. Wang to concentrate solely on his child's treatment.
Summary and Key Insights
Mr. Wang's case serves as a testament to the crucial role of early diagnosis, choosing a suitable treatment plan, and the value of cross-border medical collaboration. Through Medebound HEALTH's support, patients can access cutting-edge treatments, fostering greater optimism for their future lives. This experience underscores the importance of timely and effective medical intervention, coupled with expert guidance and comprehensive support services.
How Medebound HEALTH can assist you？
Initial Consultation: Reach out to us to discuss your needs. Our dedicated care team will concisely outline all treatment options, ensuring you understand each choice without feeling overwhelmed or stressed.
Consultation with a Specialist: We will arrange a remote video consultation with an expert from a leading U.S. hospital. Professional translation services will be provided to bridge any language gaps, facilitating clear and comfortable communication between you and the doctor regarding your condition and recovery expectations.
Tailoring Your Treatment Plan: Following the consultation, we will collaborate with you to develop a personalized treatment plan. We will meticulously explain potential treatments, their durations, costs, and expected outcomes.
Travel Assistance: Should you require travel to the U.S. for treatment, we will streamline the process for you. This includes handling visa applications, booking flights, securing comfortable accommodation near the hospital, arranging airport transfers, and making all necessary travel arrangements for your family.
Hospital Arrival Support: Upon your arrival at the U.S. hospital, our team will be there to support you every step of the way. We will facilitate communication with the doctors, explain the treatment plan, provide emotional support to your family, and leverage our long-standing relationships with U.S. hospitals to negotiate significant discounts for you—often ranging from 15% to 50%, far exceeding what an individual patient could typically negotiate.
Ongoing Support After Treatment: Our care does not end when you return home. We will continue to assist you with rehabilitation exercises, monitor your recovery progress, arrange follow-up consultations with the doctor as needed, and support you throughout your recovery journey until you achieve full recovery and meet your health goals.
Medebound HEALTH: Bridging you to the world's leading medical resources
For those seeking world-class treatment for complex medical conditions, Medebound HEALTH stands as your optimal choice. Leveraging our partnerships with leading U.S. medical institutions, we offer comprehensive support throughout your journey, whether it involves remote video consultations or traveling to the U.S. for advanced treatment options.
Contact Medebound HEALTH today and discover how we've helped thousands of families access top-tier mental healthcare.
Website: https://www.medeboundhealth.com/
Toll-Free US Number: +19173101780
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