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SMA and the new miracle drug Spinraza
Spinal muscular atrophy, or SMA, is a genetic disease that attacks nerve cells in the spinal cord. These nerve cells, known as motor neurons, communicate with the voluntary muscles in the body, such as those in the arms and legs. As more neurons are lost, muscles start to weaken and breathing, crawling, swallowing, and walking can become more difficult. It also can be difficult for those with SMA to control movements in their heads and necks. Though SMA runs in families, parents can exhibit no symptoms but still carry the gene. There are many types of SMA, and, according to the National Institute of Neurological Disorders and Stroke, the type of SMA is determined by the age of onset and the severity of symptoms. SMA Type I is evident at birth or within the first few months of a child being born. Symptoms may include difficulty swallowing, feeble movements of the arms and legs, loose limbs and trunk, impaired breathing, and a weak sucking reflex. SMA Type II is evident between the ages of six and 18 months, and the legs are typically more impaired than the arms. An early indicator may be a baby who is not crawling or walking. SMA Type III may be evident when a child reaches his or her toddler years, but sometimes does not appear until adolescence. When a child has SMA Type III, he or she may have trembling fingers and may experience difficulty getting up from a sitting position. Children with SMA Type II or Type III are at an increased risk of respiratory infections.
What causes SMA?
According to the Muscular Dystrophy Association, chromosome 5 SMA, the most common form of SMA, is caused by a deficiency of SMN, a motor neuron protein. This protein is necessary for normal motor neuron function, and a deficiency of SMN is caused by mutations on chromosome 5 in the SMN1 gene. Mutations in other genes may also cause SMA, and such cases are referred to as non-chromosome 5 SMA.
What are the symptoms of SMA?
There are various types of SMA, and the symptoms can vary depending on the type of SMA a person has. The following are some of the symptoms associated with the various types of SMA.
- Type 1: Type 1 is the most severe type of SMA. When a child is type 1, he or she may have difficulty supporting his or her head and sitting without help. Difficulty swallowing and weakness in the arms and legs may also occur. The muscles that control breathing also can be compromised when a child has type 1 SMA, and breathing problems can affect a child’s life expectancy.
- Type 2: Type 2 SMA typically affects the legs more than the arms. Type 2 SMA affects children between six and 18 months of age, and such kids may be able to sit, stand or walk with help.
- Type 3: The mildest form of SMA, type 3 may be referred to as juvenile SMA. Children with type 3 can often stand or walk without help, but they may have difficulty climbing stairs, getting up from a chair or running.
- Type 4: Type 4 SMA begins in adulthood, and men and women may experience muscle weakness, twitching or breathing problems. Physical therapy can help men and women better manage their symptoms, which typically only affect the upper arms and legs.
Recently the FDA approved an amazing new drug Spinraza to treat symptoms of SMA and prolong these life of individuals taking Spinraza. With a few injections, patients with SMA may be able to gain muscle strength and live longer by a year or more. It cost $125,000 an injection, and in the first two months one takes 4 injections in the spine. That’s half a million dollars for the first four shots. Subsequent costs for this possibly lifelong therapy are expected to be about $375,000 annually, with about three injections required each year. While this may seem extremely expensive the drug was developed to save lives, along with some resources available to help individuals requiring the drug. With coverage being more readily available to those effected the most, starting with SMA type I. Remember if you are trying to obtain Spinraza use every resource available at your disposal and educate yourself on the drug, available resources, and your healthcare. Don’t be afraid to ask questions and challenge your neurologist who would prescribe Spinraza along with your insurance representative, and if you have to, seek out a social worker for assistance. Be your own advocate. Take into account this drug has been developed and approved in a significantly short amount of time. Spinraza is for a relatively low percentage of the population, lots of money and resources were put into the development of this drug. For more information and resources go to SMAnewstoday.com. Remember to connect yourself with other individuals with SMA to share resources and connect with others who are going through the same thing you may be experiencing with SMA.
Parents can also learn more about SMA by visiting the Muscular Dystrophy Association online at www.mda.org & www.curesma.org
Live Better
When ‘Head in the Clouds’ Means Staying Ahead
(Family Features) You approve a mortgage in minutes, your medical claim is processed without a phone call and an order that left the warehouse this morning lands at your door by dinner. These moments define the rhythm of an economy powered by intelligent cloud infrastructure.
Once seen as remote storage, the cloud has become the operational core where data, AI models and autonomous systems converge to make business faster, safer and more human. In this new reality, the smartest companies aren’t looking up to the cloud; they’re operating within it.
Public cloud spending is projected to reach $723 billion in 2025, according to Gartner research, reflecting a 21% increase year over year. At the same time, 90% of organizations are expected to adopt hybrid cloud by 2027. As cloud becomes the universal infrastructure for enterprise operations, the systems being built today aren’t just hosted in the cloud, they’re learning from it and adapting to it.
Any cloud strategy that doesn’t account for AI workloads as native risks falling behind, holding the business back from delivering the experiences consumers rely on every day.
After more than a decade of experimentation, most enterprises are still only partway up the curve. Based on Cognizant’s experience, roughly 1 in 5 enterprise workloads has moved to the cloud, while many of the most critical, including core banking, health care claims and enterprise resource planning, remain tied to legacy systems. These older environments were never designed for the scale or intelligence the modern economy demands.
The next wave of progress – AI-driven products, predictive operations and autonomous decision-making – depends on cloud architectures designed to support intelligence natively. This means cloud and AI will advance together or not at all.
The Cognitive Cloud: Cloud and AI as One System
For years, many organizations treated migration as a finish line. Applications were lifted and shifted into the cloud with little redesign, trading one set of constraints for another. The result, in many cases, has been higher costs, fragmented data and limited room for innovation.
“Cognitive cloud” represents a new phase of evolution. Imagine every process, from customer service to supply-chain management, powered by AI models that learn, reason and act within secure cloud environments. These systems store and interpret data, detect patterns, anticipate demand and automate decisions at a scale humans simply cannot match.
In this architecture, AI and cloud operate in concert. The cloud provides computing power, scale and governance while AI adds autonomy, context and insight. Together, they form an integrated platform where cloud foundations and AI intelligence combine to enable collaboration between people and systems.
This marks the rise of the responsive enterprise; one that senses change, adjusts instantly and builds trust through reliability. Cognitive cloud platforms combine data fabric, observability, FinOps and SecOps into an intelligent core that regulates itself in real time. The result is invisible to consumers but felt in every interaction: fewer errors, faster responses and consistent experiences.
Consumer Impact is Growing
The impact of cognitive cloud is already visible.
In health care, 65% of U.S. insurance claims run through modernized, cloud-enabled platforms designed to reduce errors and speed up reimbursement. In the life sciences industry, a pharmaceuticals and diagnostics firm used cloud-native automation to increase clinical trial investigations by 20%, helping get treatments to patients sooner. In food service, intelligent cloud systems have reduced peak staffing needs by 35%, in part through real-time demand forecasting and automated kitchen operation. In insurance, modernization has produced multi-million-dollar savings and faster policy issuance, improving both customer experience and financial performance.
Beneath these outcomes is the same principle: architecture that learns and responds in real time. AI-driven cloud systems process vast volumes of data, identify patterns as they emerge and automate routines so people can focus on innovation, care and service. For businesses, this means fewer bottlenecks and more predictive operations. For consumers, it means smarter, faster, more reliable services, quietly shaping everyday life.
While cloud engineering and AI disciplines remain distinct, their outcomes are increasingly intertwined. The most advanced architectures now treat intelligence and infrastructure as complementary forces, each amplifying the other.
Looking Ahead
This transformation is already underway. Self-correcting systems predict disruptions before they happen, AI models adapt to market shifts in real time and operations learn from every transaction. The organizations mastering this convergence are quietly redefining themselves and the competitive landscape.
Cloud and AI have become interdependent priorities within a shared ecosystem that moves data, decisions and experiences at the speed customers expect. Companies that modernize around this reality and treat intelligence as infrastructure will likely be empowered to reinvent continuously. Those that don’t may spend more time maintaining the systems of yesterday than building the businesses of tomorrow.
Learn more at cognizant.com.
Photo courtesy of Shutterstock
SOURCE:
Cognizant
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Digital Therapeutics Offer Hope to Rural Residents
(Family Features) While mental health challenges can affect virtually anyone living anywhere, there are certain populations that are at particular disadvantages for a variety of reasons. Rural areas in the United States have an estimated 17.58 million people in nonmetropolitan areas that experienced depression in 2024, according to microdata from the National Health Interview Survey, but these areas may not have enough support and treatment options.
To learn more about rural residents’ experiences with mental health and awareness of additional or alternatives to traditional treatments that may be effective in rural areas, Rural Minds, the only national 501(c)(3) nonprofit focused on advocating for rural mental health, conducted a survey among rural Pennsylvania residents. Nearly 7 in 10 participants (69%) indicated in the past year they have either experienced symptoms of depression themselves or been concerned about someone close to them who is dealing with depression.
“While there are many effective depression treatments in use by mental health professionals, common barriers to mental health treatment for residents of rural areas include the lack of confidentiality in small communities, fewer providers and greater distance to access mental health services,” said Jeff Winton, founder and chairman of Rural Minds. “In addition, many rural residents are either uninsured or underinsured.”
The poll also showed 3 in 4 rural residents (76%) are aware of talk therapy as a depression treatment or intervention, and more than two-thirds (68%) are aware of pharmacological treatments. However, significantly less are aware of additional therapies. For example, only 17% indicated being aware of digital therapeutics and 11% reported awareness of eye movement desensitization and reprocessing.
“Depression is a significant challenge for people living in rural areas,” said Robert E. Nelson, MD, co-owner of DGR Behavioral Health, LLC, and medical director at Caron Counseling Center. “In some parts of the country, mental health services are nonexistent or very limited in rural communities. Additionally, the factors leading to depression can be different for those living outside urban and suburban communities.”
In areas where access to traditional services is limited, other options may help fill the gaps for individuals experiencing depression. For example, nearly half of survey participants (47%) would be interested in a digital app for depression as part of their treatment plan.
What are prescription digital therapeutics (PDTs)?
Technology is changing the way patients receive treatment in many aspects of medicine. That is also true when it comes to mental health. PDTs are health softwares, delivered using a device such as a smartphone or computer, to treat or alleviate a condition, disorder, disease or injury. This type of treatment may need to be paired with traditional forms of treatment to help address a variety of conditions, including mental health conditions.
Treatment delivered digitally can include brain-training exercises, cognitive behavioral therapy and behavioral reinforcement exercises. Some PDTs can also adapt based on usage.
To be considered a true prescription digital therapeutic, the software must be authorized by the Food and Drug Administration (FDA), which gives users greater confidence in its usage and outcomes.
Prescription digital therapeutics offer an additional or alternative treatment.
In addition to the potential positive implications for improving access to mental health treatment options among rural residents, there are some other potential advantages to prescription digital therapeutics:
- Less likely to cause side effects. Because there are no additional medications involved, this form of therapy doesn’t carry the same risk of medicine-related side effects or drug interactions with other medications that the user may be taking.
- Studied for safety and effectiveness. In order to qualify for FDA authorization, they are studied in clinical trials to verify their safety and effectiveness.
- Convenient. Since PDTs are provided via a personal device, such as a smartphone or tablet, the patient can access treatment when and where it’s most convenient.
- Private. The nature of a PDT means it’s delivered remotely, and patients can pursue treatment within their own homes or other private locations without the need to make regular visits to a doctor’s office.
Learn more about the mental health challenges facing Americans and find a prescription app at ruraldepressionoptions.com.
Photo courtesy of Shutterstock
SOURCE:
Rural Minds
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Finding Your Rhythm Again: Reestablishing Routines in the New Year
(Family Features) The holiday season is special, fun and often a bit chaotic, filled with family gatherings and joyful celebrations. Once the decorations come down and chaos subsides in the new year, many families face a familiar challenge: getting children back into their regular routines.
After weeks of excitement, travel and inconsistent schedules, the transition back to structure can feel daunting. However, with a thoughtful approach, parents can help their children ease back into their daily rhythms, and in the process, restore calm for the entire family. Dr. Lauren Loquasto, senior vice president and chief academic officer at The Goddard School, provides this insight and guidance for parents.
Understand Why Routines Matter
Children thrive on predictability. Routines provide a sense of security, reduce stress and support emotional regulation. Children feel safe, even proud, when they know what comes next. Routines also help them develop independence and self-control. After the holidays, reestablishing these patterns is essential for a smooth return to school and everyday life.
Start with Sleep
For young children, sleep is the foundation of a successful routine. Consistent sleep supports focus, tolerance and cooperation. For optimal brain repair and growth, preschoolers typically need 10-13 hours of sleep per night, but seasonal festivities can disrupt sleep schedules, frequently leaving children overly tired.
Begin by gradually adjusting bedtime by moving it earlier in 15-minute increments each night until you resume your regular schedule. Pair this with calming pre-bedtime rituals like reading or quiet play to signal it’s time to wind down. The morning routine is equally important. Strive for consistent wake-up times, even on the weekends.
If your child is resistant, teach them how their sleep routine helps their body and mind. For example: “Your body relaxes and grows stronger when you get enough sleep, so you feel good afterward.”
Reintroduce Standard Mealtimes
Holiday snacking and large meals at family gatherings can disrupt normal eating habits. Reinstate regular meal and snack times to help your child feel grounded. Offer balanced options and involve them in planning or preparing meals, as they’re more likely to embrace healthy choices when they have a role in the process.
Prioritize Physical Activity
After extended periods of lounging indoors – and likely an overdose of screen time – children need movement to burn energy and boost their moods. Coordinate outdoor play and family walks. If the weather isn’t cooperative, move around indoors with hide-and-seek and dance sessions. Physical activity not only supports health but also helps regulate sleep and behavior.
Strategically Provide Choices
When children have a voice, they are more likely to stay engaged and stick to their routines. As your child settles back into familiar patterns, this is an ideal moment to assess where you can ease your grip just enough to support their growing autonomy. This doesn’t mean relinquishing full decision-making power; rather, it’s about offering structured, intentional choices that help them feel a sense of control over their day.
You know your child best, so you can determine when to be flexible. For example, it may not matter if they change into their pajamas right after dinner or just before bed but shifting when they go to the bathroom could completely disrupt their routine and, in turn, your day.
Model Behavior
Children learn by example and they are always watching their parents. If you reestablish and consistently maintain your own routines – healthy meals, quality sleep and physical activity among them – your child is more likely to follow suit. Make it a family effort rather than a set of rules imposed on them, and make it fun. To help routines stick, consider sticker charts to track progress and incentivize independence and accountability.
Resetting routines isn’t about perfection; it’s about creating a supportive environment where children feel secure and prepared for what’s ahead. With patience, consistency and a positive attitude, you can help your child start the new year on the right foot.
For more parenting guidance and insights, including a blog and webinar series, visit the Parent Resource Center at GoddardSchool.com.
Photos courtesy of Shutterstock
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