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CME / ABIM MOC / CE

Best Practices in the Care of Adult Patients With Spinal Muscular Atrophy

  • Authors: Perry Shieh, MD, PhD; Sally Dunaway Young, PT, DPT; Ikjae Lee, MD; Tina Duong, PT, PhD
  • CME / ABIM MOC / CE Released: 1/20/2023
  • Valid for credit through: 1/20/2024
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  • Credits Available

    Physicians - maximum of 0.50 AMA PRA Category 1 Credit(s)™

    ABIM Diplomates - maximum of 0.50 ABIM MOC points

    Nurses - 0.50 ANCC Contact Hour(s) (0.25 contact hours are in the area of pharmacology)

    IPCE - 0.25 Interprofessional Continuing Education (IPCE) credit 

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    • ABIM MOC points

Target Audience and Goal Statement

This activity is intended for neurologists, orthopedists, orthopedic surgeons, physical therapists, nurses/nurse practitioners and primary care physicians.

The goal of this activity is for learners to be better able to identify the needs specific to adult patients with SMA, the role of key team members involved in the care of adult patients with SMA, and the impact of current therapeutics on adult patients with SMA.

Upon completion of this activity, participants will:

  • Have increased knowledge regarding the
    • Burden of disease for adult patients with SMA
    • Clinical data on the use of approved pharmacotherapies for the management of SMA in adult patients
    • Differential responsibilities relating to the care of adult patients with SMA between neurologists and advanced practice providers


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Faculty

  • Perry Shieh, MD, PhD

    Professor of Neurology and Pediatrics
    University of California Los Angeles
    Los Angeles, California

    Disclosures

    Perry Shieh, MD, PhD, has the following relevant financial relationships:
    Consultant or advisor for: Alexion; Biogen; Gene Therapies; Genentech; Novartis; Pfizer; Sarepta Therapeutics
    Speaker or member of speakers bureau for: Alexion; Argenx; Biogen; Catalyst; CSL Behring; Grifols; Genentech
    Research funding from: AMO Pharma; Astellas Gene Therapies; Biogen; Catalyst Pharmaceuticals; Fulcrum Therapeutics; Novartis Gene Therapies; Pfizer; PTC Therapeutics; ReveraGen BioPharma; Sarepta Therapeutics; Solid Biosciences

  • Sally Dunaway Young, PT, DPT

    Research Physical Therapist
    Clinical Research Manager
    Stanford University School of Medicine
    Palo Alto, California

    Disclosures

    Sally Dunaway Young, PT, DPT, has the following relevant financial relationships:
    Consultant or advisor for: Biogen Idec; Roche; Scholar Rock
    Research funding from: Biogen; Ionis Pharmaceuticals

  • Ikjae Lee, MD

    Assistant Professor of Neurology
    Columbia University Irving Medical Center
    New York, New York

    Disclosures

    Ikjae Lee, MD, has the following relevant financial relationships:
    Consultant or advisor for: Alexion

  • Tina Duong, PT, PhD

    Physical Therapist
    Director Clinical Outcomes Research & Development
    Division of Neuromuscular Medicine
    Stanford University School of Medicine
    Stanford, California

    Disclosures

    Tina Duong, PT, PhD, has no relevant financial relationships.

Editor

  • Katherine Carpenter, PhD

    Medical Education Director, WebMD Global, LLC

    Disclosures

    Katherine Carpenter, PhD, has the following relevant financial relationship:
    Advisor or consultant for: Eisai (former), GW Pharmaceuticals (former)

  • Yoji Yamaguchi, MA, ELS

    Scientific Content Manager, WebMD Global, LLC

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    Yoji Yamaguchi, MA, ELS, has no relevant financial relationships.

  • Pakinam Aboulsaoud, ​PharmD​

    Senior Medical Education Director​, Medscape, LLC

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    ​​Pakinam Aboulsaoud​, ​PharmD​, has no relevant financial relationships. 

  • Megan Breuer, PhD

    Medical Writer, Medscape, LLC

    Disclosures

    Megan Breuer, PhD, has the following relevant financial relationships:  
    Consultant/advisor for: Paratek Pharmaceuticals, Inc. (former) 
    Owns stock (publicly traded) in: Bristol Myers Squibb Company; Johnson and Johnson; Paratek Pharmaceuticals, Inc; Vertex Pharmaceuticals, Inc. 

Compliance Reviewer/Nurse Planner

  • Lisa Simani, APRN, MS, ACNP

    Associate Director, Accreditation and Compliance, Medscape, LLC

    Disclosures

    Lisa Simani, APRN, MS, ACNP, has no relevant financial relationships.

Peer Reviewer

This activity has been peer reviewed and the reviewer has disclosed no relevant financial relationships.


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Developed through a partnership between Medscape and Cure SMA.



In support of improving patient care, Medscape, LLC is jointly accredited with commendation by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.

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CME / ABIM MOC / CE

Best Practices in the Care of Adult Patients With Spinal Muscular Atrophy

Authors: Perry Shieh, MD, PhD; Sally Dunaway Young, PT, DPT; Ikjae Lee, MD; Tina Duong, PT, PhDFaculty and Disclosures

CME / ABIM MOC / CE Released: 1/20/2023

Valid for credit through: 1/20/2024

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Activity Transcript

Perry Shieh, MD, PhD: Hello, my name is Perry Shieh. I'm a neuromuscular neurologist based in Los Angeles, California. Welcome to this activity, which is titled "Best Practices in the Care of Adult Patients with Spinal Muscular Atrophy." I'm joined today by Dr Sally Dunaway Young, a research physical therapist based at Stanford University School of Medicine, and Dr Ikjae Lee, a neuromuscular specialist based at Columbia University Irving Medical Center in New York.

I'm excited about this program today because I think that the care of adults with spinal muscular atrophy (SMA) has been a topic that is underrepresented, and with new disease-modifying therapies (DMTs), we'll see more adult patients with SMA as they live longer lives on treatment. In this topic, we'll be evolving as we see new phenotypes in treated patients.

To kick things off, I'd like to turn to Ikjae. Ikjae, as a practicing adult neurologist, can you tell us what a typical adult patient with SMA is like and discuss what needs are unique to these patients?

Ikjae Lee, MD: Thank you, Dr Shieh. Let me share 2 adult SMA cases to help our audience understand the needs of our patients with SMA. Case 1. Victoria is a young adult in her mid-twenties who came to see me in the outpatient neuromuscular clinic. She was diagnosed with SMA type 2 when she was 20 months of age with homozygous SMN1 deletion and 2 SMN2 copies. She had delayed motor milestones and never achieved functional ambulation.

Her memory actually goes back to age 5, and she remembers that she was always on the wheelchair. Currently, she doesn't have movement at all in her proximal leg muscles, and very little movements left with her ankles. Her arms gradually weaken bilaterally that she can't lift her arms anti-gravity. She can still use her hands to control her power wheelchair and use computers, which provides her some degree of independence.

At night, she uses mask breathing with a bilevel positive airway pressure (BIPAP) machine, to compensate her respiratory muscle weakness. And she also uses a cough assist machine to compensate for weak cough. Her speech and swallowing are still functional, and despite of all of her functional limitations caused by the SMA, she remains positive and hopeful that the treatment will keep her stable or improve her function, which is why she came to visit us in the clinic. At the same time, she's fearful and she wishes to preserve her hand function and breathing function that are both vital for her existence.

Now, next to case 2. Jonathan is in his early thirties. He was diagnosed with SMA type 3 at age 17 with homozygous SMN1 deletion and 4 SMN2 copies. He remembers that he had difficulty with athletic activities at school. In his early adulthood, he gradually developed proximal leg weaknesses, and found to have muscle atrophy his legs, which led to genetic testing and eventually the diagnosis.

He can still walk, but he can't stand up from sitting position without using his arms and hands, and he has difficulty climbing stairs. More recently, he's noticing mild but gradual decline in arm strength, and also notice some shortness of breath with long distance walk, which made him anxious and worried, as he thought his breathing muscles would soon get weaker. He's seeking treatments that will stabilize his progression of disease and improve his daily function.

Victoria's and Jonathan's progressions of disease are consistent with the natural history of SMA in adult population. SMA types are clinically determined by age of onset and maximum motor function achieved, and also correlates with SMN2 copy numbers. More copies, milder symptoms. Among the 4 SMA types, type 2, 3, and 4 survive to adulthood without treatment; while we may see patients with SMA type 1 now survive to adulthood with treatment, respiratory and nutritional support.

Typical progression begins in the leg muscles, followed by arm, respiratory and bulbar muscle weaknesses, and proximal muscles affected earlier and more severely compared with distal muscles. Although SMA 2 patients are almost always wheelchair bound, while 50% of SMA 3 and almost SMA 4 patients will be ambulatory.

However, even in SMA 4, the mildest form, functional impairments were obvious in walking, upper body use, and breathing in the cross-sectional study by Lorenzo Maggi and colleagues, it was also demonstrated that the functional impairments are more severe in older age, supporting gradual decline in all types of SMA, which is in line with a previous 24-month longitudinal study by Georgia Coratti and colleagues showing upper limb function decline over this duration in type 2 and 3 patients with SMA.

The fear and anxiety of losing functions that are keeping them independent is the repeated theme that I hear from the patients with SMA, something we hope to address with the recent development of therapeutics.

Dr Shieh: OK. Sally, as a physical therapist specializing in the treatment of SMA, what is your take on adults with SMA?

Sally Dunaway Young, PT, DPT: Thanks, Dr Shieh. The natural history data confirm what we expect: that, in general, the progression of weakness is correlated with the age of symptom onset. The later the onset, the longer the ambulant period, as we've seen significant differences between those diagnosed with type 3A and 3B, which is symptom onset before 3 years of age and after 3 years of age.

We know muscle weakness is a key impairment, and there is a well-defined progression of muscle weakness pattern into adulthood. For those with type 2A, those able to sit, we see a steady decrease in strength and function; for those with type 2B, who are able to stand, and 3A, as I mentioned before, symptom onset before 3 years old but walking, we do see a relatively stable phase of strength followed by a pronounced decline in the third decade of life. For those diagnosed with type 3B, we do see a relatively stable phase of strength followed by a pronounced decline after age 40.

Regarding joint contractures, they do remain problematic in the adult population, with knee flexion contractures increasing steadily into adulthood, followed by hip and ankle planter flexion contractures. We also see a loss of range of motion of the shoulder, the elbow and the wrist, increasing progressively with age.

Bulbar dysfunction is a common impairment affecting SMA adults, and age has been shown to relate to reduced mandibular range of motion, primarily in SMA types 1 and 2. We also see weakness of masticatory and tongue muscles, which complicates eating and swallowing, and this can interfere with their ability to maintain body weight, perform oral hygiene, and getting dental and medical care, which can impact their safety and quality of life.

We do see a longitudinal decline of lung function in patients with SMA, and it's most pronounced during childhood and does stabilize in early adulthood, and patients with late onset, SMA types 3B and 4, are likely to have a stable lung function throughout life, with some exceptions.

Considering these impairments and their continued progression into adulthood, we must consider the functional limitations this puts on the individuals. Functionally, we see upper extremity function decline after the loss of ambulation, and the effects of cumulative yearly loss are obviously not trivial for these patients, and will eventually affect their daily functioning, making them dependent on functional abilities.

We know adults want to be independent and seek to stabilize and or improve their activities of daily living (ADLs), including self-feeding, bathing, toileting, and transferring. Adult individuals with SMA commonly report limitations in mobility and daily activities associated with deteriorating physical health, alongside emotional difficulties, fatigue, and perceived lack of societal support. However, our recent systematic review found no evidence regarding effective interventions for the adult SMA population.

Dr Shieh: Great. Well, thank you both. I think I can summarize some of the key points. Although many adults with SMA feel relatively stable, studies show that they continue to lose motor function, albeit more slowly than in the earlier phase of the disease. The progression may result in loss of clinically meaningful motor function. This includes loss of ambulation in patients who are still walking, and the loss of upper extremity function, which impacts the patient's activities of daily living. This loss results in significant morbidity and loss of quality of life. Bulbar function and respiratory function may also worsen. Contractions may develop, which may further complicate the patient's mobility.

Let's quickly review the pathophysiology of SMA. Many of you may know that SMA is an autosomal recessive genetic motor neuron disease that results in the relative deficiency of a protein known as survival motor neuron (SMN) protein. The SMN protein is primarily expressed from the SMN1 gene.

Now, there is a second gene, the SMN2 gene, which is only slightly different from the SMN1 gene, but this results in alternative splicing that more often results in an unstable non-functional protein. Patients with SMA have defective copies of the SMN1 gene, however, and thus, their SMN protein is only expressed from the SMN2 gene. And because the SMN2 gene does not express functional SMN protein efficiently, these patients have a relative deficiency of SMN protein, resulting in the progressive motor neuron degeneration that we see in these patients.

There are 2 treatments that are available for adults that are designed to enhance SMN protein expression. These both work by modulating splicing. This results in increased full-length SMN messenger RNA (mRNA), and thus, more functional SMN protein. There's also a gene therapy that is FDA approved for infants and children under 2 years of age.

Just a quick overview about these medications. Risdiplam is a small molecule. It was identified based on its ability to modulate splicing of the SMN2 gene. It's administered orally as a liquid solution, and the patients take it every day, either orally or through a gastrostomy tube. And data from randomized clinical trials are available, although in adults, they're relatively limited. There are some patients in the randomized clinical trial that are adults over the age of 15, but they're less than 25 years of age. Now, we are starting to see some real-world data in adults, although no data have been reported on adult-only populations.

Now, nusinersen is an antisense oligonucleotide that's designed to block splice enhancer sites in the SMN pre-mRNA, and this results in the modulation of the splicing that we're looking for. It is administered intrathecally. There are 4 loading doses, and there are maintenance doses every 4 months. In patients with complex spine, however, fluoroscopy and sometimes computed tomography (CT)-guided administration is necessary. There are no randomized placebo-controlled trials in adults, however, but efficacy in adults has been shown in real-world data, although this is not controlled.

On this next slide are data from the SUNFISH study. This is a double-blind placebo-controlled study of risdiplam, and what you can see here is their primary data set, which includes 180 patients, ages 2 through 25. It did show efficacy in motor function. This is the 32-item Motor Function Measure (MFM32), and you can see over the first year there was a separation of the MFM32 scores of these patients. However, only 13% of these patients were over the age of 18, so the adult population only constituted 13% of these patients.

And just as a comparison, this is a similar group of patients, similar age, at least, a natural history study looking at untreated patients ages 6 through 30, at least in this dataset. And you can see we would expect a decline among these patients. And additional data on the Revised Upper Limb Module, capturing upper limb function, as well as Hammersmith Functional Motor Scale Expanded (HFMSE) scales also showed a trend to benefit.

If you look at these functional outcome measures through the different age groups, you can see here ages 2 through 6, 6 through 11, 12 through 17 and 18 through 25, you can see that there tended to be more benefit in the younger population than the older population.

These are the adverse events (AEs) that were captured in the SUNFISH study, and you can see that the most common ones were pyrexia, diarrhea, and rash.

Now turning over to nusinersen. Nusinersen, again, there are no double-blind placebo-controlled studies [in adults], but what has been reported is real-world data that capture motor function in [adult] patients that have been treated with nusinersen. Of course, these are not randomized controlled studies, so we can only extrapolate based on what we would expect untreated patients to look like.

And over here on the right is a figure that indicates the degree of improvement on the HFMSE scores. It is summarized across 30 papers that report real-world data on patients treated with nusinersen. As you can see, you can see that most studies showed an improvement on the HFMSE score in the first year of treatment. As a comparison, you can see some of the natural history studies that were included in this meta-analysis. And what we would expect is functional decline over a similar period of time.

Summary of these studies suggest that adults that are treated with nusinersen, the mean change would increase, showing a benefit. The meta-analysis showed the average to be about 1.87 points on the HFMSE score; whereas the natural history study suggests that we would expect untreated adult patients to decline.

Now, with regard to safety, this is some of the safety information from one of the studies. This is the German study showing that the most common AEs reported were headache, back pain, and nausea. These may be procedure related.

In summary, when we're thinking about treatment for adults with SMA, we should start thinking about the mechanism of action, and the fact that this is really realistically only going to preserve motor neurons. The realistic effects on disease modification would be stabilization of motor function. We would only expect SMN-restoring therapies to prevent further motor neuron degeneration. Significant gains in motor function might be seen, but they're generally modest.

The goals of treatments should therefore be specific to the patient's disease state. For example, a non-ambulatory patient may have some modest gains in overall motor function, but realistically, the patient's goals should be to stabilize muscles, perhaps facial muscles, upper limb function, and axial muscle strength.

At this point, Ikjae, Sally, I don't know if you have any additional comments on the clinical data that we've presented so far.

Dr Lee: I think the clinical data are definitely encouraging. However, we should recognize the limitation that we don't have a lot of data in the adults, especially in the older population. We have to discuss this limitation with the patient when talking about the therapeutic options.

Dr Shieh: Great. And Sally, perhaps you can comment on the need for physical therapy (PT) and how that changes with DMT.

Dr Dunaway Young: Thanks, Dr Shieh. Our rehabilitation goals don't necessarily change for this population, as we still want to focus on maintaining range of motion and strength, review contracture management, and prevent or limit contractures from further developing with stretching and bracing. We also have the goal to help them manage pain and edema, utilizing different modalities.

We want them to optimize function by utilizing adaptive equipment and assistive technology. We want to advise them on fatigue and energy conservation, help foster participation, and help them anticipate any complications or problems that may occur throughout the years. We want to support access to treatment, as well.

In the era of DMTs, we should be putting a greater emphasis on individuals meeting the standard-of-care recommendations for exercise and interventions. We want to educate these individuals to try and maximize any potential therapeutic treatment benefit. They may be beginning, so we want to promote exercise programs and activities that encourage muscle activation, which can have an effect on maintaining and improving function, strength, range of motion, endurance, balance, activities of daily living, and participation in their school, social activities, or occupation.

Dr Shieh: In summary, I think we should really keep in mind that the current data on efficacy and safety of risdiplam and nusinersen and adults with SMA is limited and based mostly on real-world, post-FDA approval data. Although limited, these data have shown favorable results following treatment with risdiplam and with nusinersen. Consideration of treatment and follow-up to determine benefit should be on a case-by-case basis, and close monitoring and follow-up are essential to learning how SMA treatments can be best utilized in adults with SMA.

Now, I'm going to shift to multidisciplinary care, and it's essential that good care of our adult patients is provided by coordinating among many health professionals who are involved. Let's talk about this, as well. Ikjae, let's start with you. Why don't you tell us a little bit about your view on the role of neurologists in this multidisciplinary care?

Dr Lee: Sure. The role of a neurologist, frequently a neuromuscular specialist, is to establish diagnosis, provide DMTs, and coordinate multidisciplinary care. First step and important step is to establish the diagnosis. History of bilateral painless progression of weakness in the legs followed by arms, and then neurological examination findings of muscle weakness, muscle atrophy, reduced reflexes in young adults should raise concern for SMA.

Genetic counseling followed by genetic testing would confirm the diagnosis with homozygous deletions or mutations in the SMN1 gene. Confirmation of SMN2 copy numbers will be informative for prognosis and will further support the prescription of medications. Once the diagnosis is established, next step is to make shared decisions with the patient on the treatment. Understanding the evidence from the clinical trials and observational studies will be really critical at this step and help make the right decision that is most beneficial for the patient.

I typically inform the patients of FDA-approved medications, such as risdiplam and nusinersen, their efficacy and side-effect profiles identified in the trials. I also set expectations, as mentioned previously. In adult patients with SMA, we might not be expecting a lot of functional improvement but may focus on stabilizing the current functions.

Starting the medication would typically involve completing a benefit investigation form, obtaining prior authorization, and if coverage is denied by the insurance  appeal process. And coverage denial for initiation of medications is not infrequent in my experience, due to insufficient data on adults over age 25. Baseline and follow-up assessments of the functional impairments and disabilities through scales such as MFM32, will be helpful in following the progression of the disease, and also documenting benefits of treatments.

Patients with SMA are best cared, in my mind, in the multidisciplinary clinic setting, to address their needs for durable medical equipment, such as wheelchairs or non-invasive ventilators, rehabilitation needs, nutritional support, social work, and sometimes even non-neurological symptom management. It is really a team effort and it's important to stay connected with multiple providers, including physical therapists, physical medicine and rehabilitation (PM&R)  physicians, pulmonologists, nutritionists, primary care doctors, nurse practitioners, and social workers in combination to provide the best quality care to our patients with SMA.

Dr Shieh: All right. Sally, what's your view of multidisciplinary care? Do you have any comments about how all the practitioners can work together?

Dr Dunaway Young: Yes, thank you. I completely agree with Dr Lee. The multidisciplinary approach really needs to continue from the transition from pediatric to adulthood, and the standard of care for SMA document updated in 2018 really highlights the additional advanced practitioners required.

Although we do know that adult individuals with SMA can have more complex needs, I think it's important to note that DMTs do not replace the team, and in fact, should encourage us to take a more comprehensive approach in educating the patient on their anticipated needs across their lifespan.

As Dr Lee just reviewed, the role of neurologist and the providers that he closely works with in managing his patients, I'd also just like to add that you also have to work with interventional radiologists to help with drug administration. Social workers have been critical, as a lot of these adult patients haven't been in the healthcare system for years, and so they're needing a lot of support to navigate insurance while seeking treatments. Help with health insurance planning, advising young adults who are able to stay on their parents' policy until 26 years old, as well as helping them navigate social security disability benefits and Medicare.

For the rehabilitation providers, as you mentioned, I think it's critical that we still stay involved for their muscle health, focusing on stretching, bracing, exercise regimens, helping address pain, contractures, mobility, ADLs, any challenges that they're experiencing in their daily life, as well as the durable medical equipment and really needing providers that do have expertise in neuromuscular care. For speech language pathologists, dysphagia and dysarthria can be addressed as well for these adult individuals with SMA.

Our role as rehab providers are really to provide consultation, evaluation, instruct and educate patients and families, help implement the standard of care, administer both functional and patient reported assessments to help track disease progression over time, and also provide proactive and intensive patient management to enhance any potential benefit they may get from a pharmacological treatment. We also advocate for them in the community, in their workplace, and just provide as much support and education as we can.

Rehab providers often work closely with orthopedic doctors and help management of their bone health. We continue to monitor into adulthood scoliosis and chest deformities, contractures, as I mentioned before, hip instability and pain, as well as fractures. They also note their vitamin D deficiency and monitor as well as potentially doing bone density (DXA) scans and providing supplementation.

Gastrointestinal, nutrition and dietician needs are also crucial, as these patients often have difficulty maintaining an optimal weight. They also need to work with speech language pathologists and potentially obtain swallow studies. We know gastrointestinal dysfunction is common. G-tube placements may be required. As they grow into adulthood, may be at risk for diabetes and doing body mass index (BMI) monitoring, resting energy expenditure, and even discussing bowel health with them.

From the pulmonary perspective and looking at their respiratory health, pulmonary function testing needs to be assessed. Non-invasive ventilation may be prescribed. They may need to discuss acute therapies and continued management, looking at sleep studies, airway clearance, and immunizations. For cardiology, the risk for hypertension, leg swelling, and the sequelae of developing progressing scoliosis. For acute care needs, the discussion of palliative care, advanced directives, as well as acute infections, including COVID.

Reproductive health is another provider that is necessary to bring in for this adult population; an OBGYN, to obtain regular exams, if they have questions about pregnancy, childbirth, or treatments, or how treatments may affect reproduction, and loss of abilities that may occur after pregnancy. I mentioned earlier dental health, and tolerance for procedures, positioning, and how to tolerate the anesthetic, as these patients may also have reduced mouth openings, making access for cleaning limited.

We also need to discuss school and work accommodations with them, needing to make sure that it's meeting ADA standards. There's a financial burden of disease, and the costs that are anticipated, especially with obtaining durable medical equipment and caregiver support and missed work. We also needed to support them with psychological health, mental health and wellbeing, and coming to terms with obtaining treatment and how to define oneself. And lastly, I think it's important to discuss sex and relationships and helping them navigate intimacy with disability.

The networking and connections we can make as providers to best facilitate these referrals will help us to be proactive and anticipate the additional needs adult individuals have and that they're experiencing as they continue to live longer. We should discuss quality of life and goals with the patients to form a collaborative care plan and encourage them to have self-advocacy.

Dr Shieh: Great. Thank you both for that thorough discussion. Obviously, multidisciplinary care is a complex topic, and so I'll just make some broad-stroke summary statements. Multidisciplinary care is needed to address the complex and unique needs of patients with SMA, particularly adults. And with DMTs now available, there will be more adult patients with SMA who need multidisciplinary care. Transition of care to adult neuromuscular specialists will be needed to adapt to the evolving landscape of neuromuscular patients.

To conclude today's discussion, adults with SMA have unique needs, and there is evidence- based data to support the use of nusinersen and risdiplam in adults. The multidisciplinary care of adults with SMA is evolving to incorporate this growing population.

Thank you, Sally, Ikjae, for your participation in this program and your insightful comments. And I thank you, the audience, for your attention. Please continue to answer the questions that follow and complete the evaluation.

This transcript has not been copyedited.

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