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Rare Disease Treatment in the USA: How UCLA's Expertise Changed Baby Wang's SMA Journey

Updated: Dec 12

For families seeking Pediatric Care in America, finding the right expertise is crucial. Just like a recent case where a Thai child found breakthrough treatment at a leading US hospital, Baby Wang's journey shows why many Travel to the USA for Medical Treatment.


The joy of welcoming a new baby quickly turned to deep concern when Mr. Wang noticed his 5-month-old son had no strength in his legs. Like many patients who visit top hospitals in the USA, the Wang family's search for answers led them to America's advanced medical system.


Similar to how Mayo Clinic transformed a cirrhosis patient's life through expert care, and how MD Anderson provided hope for Ms. Lin's rare cancer, UCLA stands among the best hospitals in the USA for complex pediatric cases. For those considering medical tourism in the USA, understanding these success stories provides valuable guidance.


What followed was a series of increasingly worrying medical tests, culminating in a genetic test that revealed the devastating news: his son had spinal muscular atrophy (SMA) Type 2, with homozygous deletion of exon 7 and exon 8 of the SMN1 gene. This diagnosis came with no effective treatment options.


At Medebound HEALTH, recognized as a leading medical facilitator for the USA with expertise in international medical care for children, we understand the urgency these families face. Our network connects patients who travel abroad for treatment with the top specialists across 72 medical specialties.


For those asking "How much does it cost to seek medical treatment in the USA?", understanding the expenses at top US children's hospitals is crucial. Our Services for facilitating medical treatment abroad include comprehensive cost analysis and planning.


Through consultation with American specialists, families gain access to innovative treatments. As experienced providers of expert pediatric care in America, we ensure seamless coordination between international medical teams.




Understanding SMA's Impact

SMA is an autosomal recessive neuromuscular disease that affects the spinal cord's anterior horn neurons.


For children like Mr. Wang's son, this means progressive muscle weakness affecting crucial functions - from crawling and walking to breathing and swallowing.


Type 2 SMA typically appears between 6-12 months, making early intervention crucial.


"When we received the diagnosis, I knew time was our enemy," Mr. Wang shares. "Each day without treatment meant potential loss of motor function. We needed answers fast."


The Path to Hope

Instead of immediately arranging travel, Medebound HEALTH took a strategic approach.


Our medical service team first organized a remote consultation to:

  • Assess Treatment Urgency And Options.

  • Explore Available US Treatments And Clinical Trials.

  • Evaluate Potential Costs.

  • Understanding Expected Outcomes.

  • Compare US Hospital Options.

We connected Mr. Wang with Mount Sinai Hospital's leading experts - Dr. Steven M. Wolf, a renowned child neurologist, and Deputy Director Patty McGoldrick. Dr. Wolf, an associate professor of neurology and director of pediatric epilepsy neuromuscular disease, brought decades of specialized experience to the case.



Dr. Steven. M. Wolf
Dr. Steven. M. Wolf is a nationally renowned child neurologist specializing in the diagnosis and treatment of pediatric neurology. Dr. Wolf is currently an associate professor of neurology at Mount Sinai Hospital and director of pediatric epilepsy neuromuscular disease/pediatric neurodevelopmental disorders. He received his medical degree from Albany Medical Center, studied child neurology at Albert Einstein College of Medicine, and is certified in pediatric neurology, clinical neurophysiology, and neurological special identification by the American Board of Pediatrics.

After fully understanding the child's condition, Dr. Wolf and Deputy Director McGoldrick told Mr. Wang that Nusinersen (trade name Spinraza, manufactured by Biogen) can be used to treat SMA in the United States.


Breaking Through with Nusinersen

When you go to the USA for treatment, accessing leading children's hospitals USA like UCLA can transform outcomes. According to the rankings of American hospitals, UCLA consistently leads in pediatric care.


The experts recommended Nusinersen (Spinraza), an FDA-approved treatment since December 2016. 


This revolutionary drug, administered through intrathecal injection, works by increasing SMN protein levels.


Clinical trials showed 40% of patients achieving improved motor function - results unseen in control groups.


The treatment plan included comprehensive care addressing:

  • Nutrition management.

  • Respiratory function monitoring.

  • Potential scoliosis prevention.

  • Regular follow-up evaluations.

  • Physical and occupational therapy.

UCLA MEDICAL CENTER

After comparing multiple institutions including Boston Children's Hospital and Seattle Children's Hospital, we recommended UCLA Medical Center - ranked #4 nationally and #1 in California - for its optimal combination of expertise and cost-effectiveness.


Making It Happen

From the moment the family landed in the US, our support was comprehensive:

  • Airport pickup service.

  • Dedicated translator.

  • Private transportation.

  • Housing arrangements.

  • Cultural orientation support.


At UCLA, Mr. Wang faced a choice between clinical trials (under $138,000) and new treatment (several thousand dollars).


Our team helped explain both options, considering long-term implications and success rates.

Within a week, treatment began.

Treatment area for 5 month old baby

"The difference in medical care was striking," Mr. Wang notes. "In America, doctors take time to answer every question. This attention to detail gave us confidence."



Results That Matter:

Like other families who travel overseas for medical treatment, the Wangs discovered that American healthcare offered unique advantages. Through our role as agencies providing services for seeking medical treatment in the USA, we ensured they received optimal care.


Treatment Progress

By January 2018, Mr. Wang's son had completed three successful Nusinersen injections at UCLA Medical Center, with the fourth treatment scheduled. "Each injection brought new hope," shares Mr. Wang. "We could see small but meaningful improvements in our son's strength."

Quality of Care Difference

The contrast in medical care was striking, doctor visits felt rushed, with barely time for questions," Mr. Wang explains. "At UCLA, doctors took time to explain everything. They wouldn't move forward until all our concerns were addressed - this level of care made a real difference in our confidence."

As a leading medical tourism agency in the United States, we understand that American medical care for kids sets global standards. The Wang family's experience at UCLA, like successful cases at Mayo Clinic and other top hospitals in the USA, demonstrates the value of accessing US healthcare.

Family's Determination

"My wife became our family's quiet hero," Mr. Wang says with emotion. "Despite the physical and mental challenges of caring for our son in a foreign country, her dedication never wavered. Together with Medebound HEALTH's support, we found strength we didn't know we had."

Mr. Wang's child, accompanied by his mother, is waiting for blood to be drawn for relevant examinations

Cost-Effective Care

Through Medebound HEALTH's strategic planning and hospital selection process, the family saved over $28,000 while accessing world-class treatment. "They compared options across multiple US hospitals," notes Mr. Wang. "UCLA offered the best combination of expertise and cost-effectiveness."

Long-Term Impact

Today, Mr. Wang shares his story to help other families facing similar challenges. "Finding the right medical partner changed everything," he reflects. "With Medebound HEALTH's guidance and UCLA's expertise, we're seeing our son progress in ways we never thought possible."

The Medebound HEALTH Difference:

  • Comprehensive pre-travel preparation.

  • 24/7 bilingual support.

  • Complete medical coordination.

  • Cost-effective treatment planning.

  • Long-term care strategy.

The Power of Second Opinions

Sometimes, you don't need to travel to get top US medical advice. Medebound HEALTH's second opinion service lets you:

Consult Top US Doctors From Home.

Save On Travel Costs.

Make Informed Decisions About Your Treatment.

Access Specialty Director-Level Professors Directly.

Get Follow-Up Care And Potential Access To Clinical Trials.

teleconsultation with the help of medebound HEALTH

Why Medebound HEALTH is Different 

Medebound HEALTH is an American overseas medical service agency established in New York. It's not an ordinary medical intermediary. 

Compared with other overseas medical service agencies, Medebound HEALTH has the following special features:

1. Direct Contact With Doctors. 

2. Crucial Relationships. 

3. Speed Advantage. 

4. Save Money. 

5. Professional Customization. 

6. U.S. Recognition. 

7. Direct Provider Model. 

8. Focus On U.S. Healthcare / Headquartered In New York. 

Take Action Today

Don't wait - every day counts. Contact Medebound HEALTH now:

Toll-Free US Number: +19173101780 

Let us connect you with the USA's top 1% of doctors across 72 specialties, whether through video consultation or treatment in the US. Our team is ready to help you access world-class care at manageable costs.

One simple message could change your treatment journey.

Contact us now, and let us guide you to the care you deserve.



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We know top doctors make a difference. Our consulting doctors are world-class physicians recognized by prestigious awards such as Castle Connolly Top Doctors, encompassing 70+ medical disciplines representing the Top 1% doctors of in the nation.

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