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One Woman’s Path to Finding Treatment for Narcolepsy

Wendy’s childhood cataplexy led to a narcolepsy diagnosis after years of excessive sleepiness. She now effectively manages her symptoms with the LUMRYZ medication.

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Wendy was compensated by Avadel Pharmaceuticals for her time. Individual results may vary.

(Family Features) For Wendy, playfully jumping on the bed as a child led to panic when, suddenly, her limbs ceased to function and she dropped to the floor. Although she needed help, she couldn’t use her voice. This was Wendy’s first experience with cataplexy, a sudden period of muscle weakness that can be triggered by strong emotions, like laughter, excitement, or anger – a hallmark symptom of narcolepsy type 1.

Narcolepsy is a complex, lifelong sleep disorder that includes symptoms like cataplexy and excessive daytime sleepiness and is estimated to affect 1 in 2,000 Americans.

Wendy’s childhood experience with cataplexy was followed by years of struggling with excessive daytime sleepiness (EDS) and trouble sleeping through the night. Her sleepiness, however, was dismissed for much of her life – often being told she was tired simply due to being busy.

“No one believed me when I said that something felt wrong,” Wendy said.

For people with narcolepsy, the lines between being asleep and awake are blurred. Instead of a natural sleep pattern, they often experience short periods of poor-quality sleep throughout the day and night, rather than restorative, consolidated sleep.

25 years after her first cataplexy attack, Wendy experienced a second episode while driving and was rushed to the emergency room.

“I stayed in the hospital for a week,” said Wendy. “Doctors first thought I likely had an attack similar to a stroke or a severe migraine. It wasn’t until I met with a neurologist and shared that I slept too much that I was referred for a sleep study and finally diagnosed with narcolepsy.”

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After receiving the right diagnosis, the next challenge for Wendy was finding the right treatment.

Her doctors first prescribed several stimulants, which didn’t alleviate her daytime sleepiness. She was then prescribed a sodium oxybate, a treatment used for EDS and cataplexy, however this particular medication required waking up in the middle of the night to take a second dose, which Wendy found herself struggling to do.

Wendy turned to the narcolepsy community, where she learned about a once-nightly medication, LUMRYZ® (sodium oxybate) for extended-release oral suspension, CIII.

LUMRYZ is the first and only U.S. Food and Drug Administration approved once-at-bedtime sodium oxybate treatment for cataplexy or EDS in adults with narcolepsy, and a medication that Wendy says she is grateful for.

LUMRYZ has a boxed warning as a central nervous system depressant and for its potential for abuse and misuse. LUMRYZ is available only through a restricted program under a Risk Evaluation and Mitigation Strategy called the LUMRYZ REMS. Most common adverse reactions (incidence ≥ 5% and greater than placebo) reported for all doses of LUMRYZ combined were nausea, dizziness, bedwetting, headache and vomiting. Please see additional Important Safety Information, including Boxed Warning below.

“When I first heard about LUMRYZ being once-at-bedtime, I was ecstatic there was an option that might help with my cataplexy and EDS without having to wake in the middle of the night for a second dose,” said Wendy.

Today, Wendy has discovered a treatment that is right for her and helps improve her EDS and cataplexy symptoms, individual results may vary.

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“My advice to others with narcolepsy is to be outspoken about your experience and learn as much as you can to be well-informed. I want to see people be diagnosed earlier, advocate for themselves, and find a treatment that’s right for them.”

If you are struggling with excessive daytime sleepiness, ask your healthcare provider about narcolepsy, and if you have been diagnosed, ask your physician if LUMRYZ is right for you. Learn more at www.lumryz.com.

Photo caption: Wendy, a person with narcolepsy

INDICATIONS
LUMRYZ (sodium oxybate) for extended-release oral suspension is a prescription medicine used to treat the following symptoms in adults with narcolepsy:

  • sudden onset of weak or paralyzed muscles (cataplexy)
  • excessive daytime sleepiness (EDS)

IMPORTANT SAFETY INFORMATION

WARNING: Taking LUMRYZ™ (sodium oxybate) with other central nervous system (CNS) depressants such as medicines used to make you fall asleep, including opioid analgesics, benzodiazepines, sedating antidepressants, antipsychotics, sedating anti-epileptic medicines, general anesthetics, muscle relaxants, alcohol, or street drugs, may cause serious medical problems, including trouble breathing (respiratory depression), low blood pressure (hypotension), changes in alertness (drowsiness), fainting (syncope), and death. The active ingredient of LUMRYZ (sodium oxybate) is a form of gamma hydroxybutyrate (GHB), a controlled substance. Abuse or misuse of illegal GHB alone or with other CNS depressants (drugs that cause changes in alertness or consciousness) have caused serious side effects. These effects include seizures, trouble breathing (respiratory depression), changes in alertness (drowsiness), coma, and death. Call your doctor right away if you have any of these serious side effects. Because of these risks, LUMRYZ is available only by prescription and filled through certified pharmacies in the LUMRYZ REMS program. You must be enrolled in the LUMRYZ REMS to receive LUMRYZ. Further information is available at www.LUMRYZREMS.com or by calling 1-877-453-1029.

It is not known if LUMRYZ is safe and effective in people less than 18 years of age.

Do not take LUMRYZ if you take other sleep medicines or sedatives (medicines that cause sleepiness), drink alcohol, or have a rare problem called succinic semialdehyde dehydrogenase deficiency.
Keep LUMRYZ in a safe place to prevent abuse and misuse. Selling or giving away LUMRYZ may harm others and is against the law. Tell your doctor if you have ever abused or been dependent on alcohol, prescription medicines, or street drugs.

Anyone who takes LUMRYZ should not do anything that requires them to be fully awake or is dangerous, including driving a car, using heavy machinery, or flying an airplane, for at least six (6) hours after taking LUMRYZ. Those activities should not be done until you know how LUMRYZ affects you.

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Falling asleep quickly, including while standing or while getting up from the bed, has led to falls with injuries that have required some people to be hospitalized.

LUMRYZ can cause serious side effects, including the following:

  • Breathing problems, including slower breathing, trouble breathing, and/or short periods of not breathing while sleeping (e.g., sleep apnea). People who already have breathing or lung problems have a higher chance of having breathing problems when they take LUMRYZ.
  • Mental health problems, including confusion, seeing or hearing things that are not real (hallucinations), unusual or disturbing thoughts (abnormal thinking), feeling anxious or upset, depression, thoughts of killing yourself or trying to kill yourself, increased tiredness, feelings of guilt or worthlessness, and difficulty concentrating. Tell your doctor if you have or had depression or have tried to harm yourself. Call your doctor right away if you have symptoms of mental health problems or a change in weight or appetite.
  • Sleepwalking. Sleepwalking can cause injuries. Call your doctor if you start sleepwalking.

Tell your doctor if you are on a salt-restricted diet or if you have high blood pressure, heart failure, or kidney problems. LUMRYZ contains a lot of sodium (salt) and may not be right for you.

The most common side effects of LUMRYZ in adults include nausea, dizziness, bedwetting, headache, and vomiting. Your side effects may increase when you take higher doses of LUMRYZ. LUMRYZ can cause physical dependence and craving for the medicine when it is not taken as directed. These are not all the possible side effects of LUMRYZ.

For more information, ask your doctor or pharmacist. Call your doctor for medical advice about side effects.

You are encouraged to report negative side effects of prescription drugs to the FDA.
Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

Please see full Prescribing Information, including BOXED Warning, and Medication Guide.

PM-US-AVGEN-0169-v2 07/2024

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SOURCE:
Avadel

Our Lifestyle section on STM Daily News is a hub of inspiration and practical information, offering a range of articles that touch on various aspects of daily life. From tips on family finances to guides for maintaining health and wellness, we strive to empower our readers with knowledge and resources to enhance their lifestyles. Whether you’re seeking outdoor activity ideas, fashion trends, or travel recommendations, our lifestyle section has got you covered. Visit us today at https://stmdailynews.com/category/lifestyle/ and embark on a journey of discovery and self-improvement.


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Lifestyle

Clinical Trials Expand Patients’ Treatment Options

Clinical trials play a crucial role in developing effective treatments for chronic health conditions. They evaluate safety and efficacy, but low enrollment delays drug approvals, limiting patient options.

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Clinical Trials

(Family Features) For health conditions with no cure, effective treatments are the next best option. However, in order to make medical interventions including new drugs, devices, vaccines and lifestyle modifications available, they must undergo a thorough evaluation process to demonstrate their benefits.

Clinical trials are an important part of this process because they provide data for researchers on the effectiveness of various treatments and interventions. However, many myths about clinical trials deter people from enrolling. In fact, more than 70% of clinical trials are delayed at least one month due to unfulfilled enrollment, according to the “Journal of Medical Internet Research.”

Delays create barriers to the drug approval process and result in fewer available treatments for chronic conditions like Crohn’s disease and ulcerative colitis. The experts at the Crohn’s & Colitis Foundation offer these facts about the importance of clinical trials:

  1. More patients can benefit from clinical trials While hundreds of participants may take part in a trial, the results can lead to new care and treatment options for an entire patient community.
     
  2. Clinical trials lead to better and improved medications. For a new treatment to be approved, it needs to demonstrate a meaningful benefit to patients, such as improved outcomes, treating a wider range of symptoms, fewer side effects or more convenience for patients.
     
  3. They provide information. Clinical trials provide evidence of a treatment’s effectiveness and safety that can be used to guide patient care.
     
  4. Trials test safety and efficacy. Clinical trials are conducted in four phases. During the first phase, the main objective is to assess the safety of a new treatment. Experimental treatments only advance to the next stages if they pass certain safety and efficacy standards
    .
  5. They help prevent bias. In a randomized controlled trial, the effects of treatment with a new drug are compared with that of a control group – either a placebo (a treatment that looks identical to the new drug but contains no active medicine) or the current standard of care. Treatment assignments are “randomized,” which means neither the trial doctors nor the participants choose which treatment each participant will receive.

How Clinical Trials Work
Before a clinical trial begins, a research team develops a clinical trial protocol. This is the research plan that describes who can join the trial, which treatments the participants will receive, what questions the researchers will try to answer, how long the trial will last and how participant privacy and safety will be protected.

Conducting clinical research relies on volunteer trial participants. Those interested in joining a clinical trial meet with clinical research professionals to discuss the trial, treatments, eligibility requirements and expectations.

Joining a Trial
If you are interested in participating in a clinical trial, do some research. Your doctor may not be aware of or proactively tell you about trials you may be eligible to join. If you find a clinical trial you may be interested in joining, discuss the trial with your doctor and other health care professionals on your research team.

Foundations supporting chronic conditions can be a good source of information. For example, you can learn more about trials affecting individuals with inflammatory bowel disease by exploring the Clinical Trials Community at crohnscolitisfoundation.org.

Photo courtesy of Shutterstock

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SOURCE:
Crohn’s & Colitis Foundation


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Why community pharmacies are closing – and what to do if your neighborhood location shutters

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Photo by Pixabay on Pexels.com

Lucas A. Berenbrok, University of Pittsburgh; Michael Murphy, The Ohio State University, and Sophia Herbert, University of Pittsburgh

Neighborhood pharmacies are rapidly shuttering.

Not long ago, Walgreens, one of the nation’s biggest pharmacy chains, announced plans to close 1,200 stores over the next three years. That’s part of a larger trend that has seen nearly 7,000 pharmacy locations close since 2019, with more expected in the coming years.

Many community pharmacies are struggling to stay open due to an overburdened workforce, shrinking reimbursement rates for prescription drugs and limited opportunities to bill insurers for services beyond dispensing medications.

As trained pharmacists who advocate for and take care of patients in community settings, we’ve witnessed this decline firsthand. The loss of local pharmacies threatens individual and community access to medications, pharmacist expertise and essential public health resources.

The changing role of pharmacies

Community pharmacies – which include independently owned, corporate-chain and other retail pharmacies in neighborhood settings – have changed a lot over the past decades. What once were simple medication pickup points have evolved into hubs for health and wellness. Beyond dispensing prescriptions, pharmacists today provide vaccinations, testing and treatment for infectious diseases, access to hormonal birth control and other clinical services they’re empowered to provide by federal and state laws.

Given their importance, then, why have so many community pharmacies been closing?

There are many reasons, but the most important is reduced reimbursement for prescription drugs. Most community pharmacies operate under a business model centered on dispensing medications that relies on insurer reimbursements and cash payments from patients. Minor revenue comes from front-end sales of over-the-counter products and other items.

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However, pharmacy benefit managers – companies that manage prescription drug benefits for insurers and employers – have aggressively cut reimbursement rates in an effort to lower drug costs in recent years. As a result, pharmacists often have to dispense prescription drugs at very low margins or even at a loss. In some cases, pharmacists are forced to transfer prescriptions to other pharmacies willing to absorb the financial hit. Other times, pharmacists choose not to stock these drugs at all.

And it’s not just mom-and-pop operations feeling the pinch. Over the past four years, the three largest pharmacy chains have announced plans to close hundreds of stores nationwide. CVS kicked off the trend in 2021 by announcing plans to close 900 pharmacy locations. In late 2023, Rite Aid said that thousands of its stores would be at risk for closure due to bankruptcy. And late in 2024, Walgreens announced its plans to close 1,200 stores over the next three years.

To make matters worse, pharmacists, like many other health care providers, have been facing burnout due to high stress and the lasting effects of the COVID-19 pandemic. At the same time, pharmacy school enrollment has declined, worsening the workforce shortage just as an impending shortfall of primary care physicians looms.

Why pharmacy accessibility matters

The increasing closure of community pharmacies has far-reaching consequences for millions of Americans. That’s because neighborhood pharmacies are one of the most accessible health care locations in the country, with an estimated 90% of Americans living within 5 miles of one.

However, research shows that “pharmacy deserts” are more common in marginalized communities, where people need accessible health care the most. For example, people who live in pharmacy deserts are also more likely to have a disability that makes it hard or impossible to walk. Many of these areas are also classified as medically underserved areas or health professional shortage areas. As pharmacy closures accelerate, America’s health disparities could get even worse.

So if your neighborhood pharmacy closes, what should you do?

While convenience and location matter, you might want to consider other factors that can help you meet your health care needs. For example, some pharmacies have staff who speak your native language, independent pharmacy business owners may be active in your community, and many locations offer over-the-counter products like hormonal contraception, the overdose-reversal drug naloxone and hearing aids.

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You may also consider locations – especially corporate-owned pharmacies – that also offer urgent care or primary care services. In addition, most pharmacies offer vaccinations, and some offer test-and-treat services for infectious diseases, diabetes education and help with quitting smoking.

What to ask if your pharmacy closes

If your preferred pharmacy closes and you need to find another one, keep the following questions in mind:

What will happen to your old prescriptions? When a pharmacy closes, another pharmacy may buy its prescriptions. Ask your pharmacist if your prescriptions will be automatically transferred to a nearby pharmacy, and when this will occur.

What’s the staffing situation like at other pharmacies? This is an important factor in choosing a new pharmacy. What are the wait times? Can the team accommodate special situations like emergency refills or early refills before vacations? Does the pharmacist have a relationship with your primary care physician and your other prescribers?

Which pharmacies accept your insurance? A simple call to your insurer can help you understand where your prescriptions are covered at the lowest cost. And if you take a medication that’s not covered by insurance, or if you’re uninsured, you should ask if the pharmacy can help you by offering member pricing or manufacturer coupons and discounts.

What are your accessibility needs? Pharmacies often offer services to make your care more accessible and convenient. These may include medication packaging services, drive-thru windows and home delivery. And if you’re considering switching to a mail-order pharmacy, you should ask if it has a pharmacist to answer questions by phone or during telehealth visits.

Remember that it’s best to have all your prescriptions filled at the same pharmacy chain or location so that your pharmacist can perform a safety check with your complete medication list. Drug interactions can be dangerous.

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Community pharmacies have been staples of neighborhoods for more than a century. Unfortunately, current trends in pharmacy closures pose real threats to public health. We hope lawmakers address the underlying systemic issues so more Americans don’t lose access to their medications, health services and pharmacists.

Lucas A. Berenbrok, Associate Professor of Pharmacy and Therapeutics, University of Pittsburgh; Michael Murphy, Assistant Professor of Pharmacy Practice and Science, The Ohio State University, and Sophia Herbert, Assistant Professor of Pharmacy, University of Pittsburgh

This article is republished from The Conversation under a Creative Commons license. Read the original article.

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A How-To Guide for Participating in Clinical Trials

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(Family Features) Clinical trials help researchers studying chronic conditions answer important questions about the diseases and their treatment options. However, uncertainty about what to expect and a lack of knowledge about how to get started can prevent patients from joining a clinical trial.

Choosing to take part in a clinical trial means helping a study team figure out if a new method of diagnosis, treatment or prevention is effective. If you live with a chronic condition, such as Crohn’s disease or ulcerative colitis, and want to help find answers for others who share your experience, a clinical trial is an option to consider.

Once you identify a study that interests you, you’ll want to talk with the professionals involved in your ongoing treatment, a clinical research coordinator and your family to gather information necessary to determine whether the clinical trial is a good fit.

To find additional information about clinical trials and begin exploring trials in your area, visit crohnscolitisfoundation.org, and consider these steps for participating in a trial.

Clinical Trials

1. Talking with Your Doctor
Your gastroenterologist and other care providers can help determine whether a clinical trial is right for you and may be able to help point you toward recommended trials. It’s important to ask if or how your doctor will continue to be involved in your care if you participate in a trial.

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2. Finding a Study
If you need help beyond your care team in identifying clinical trial opportunities in your area, organizations dedicated to your condition can be a good resource. For example, the Crohn’s & Colitis Foundation offers an online Clinical Trial Finder for individuals with inflammatory bowel disease.

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3. Talking with the Research Coordinator
A clinical trial research coordinator can provide details specific to your circumstances and needs. You can discuss potential benefits and risks, why the trial is being conducted and who is involved in the health care team. You can talk about past treatments and how this study may differ from your previous experiences. Other questions you might ask include what your options are if the trial doesn’t work, any costs you might expect and what your personal commitment will be.

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4. Evaluating the Fit for You
Once you have the necessary information, you’ll be able to consider whether you’re ready to move forward with registering for the trial. You’ll want to weigh factors like your time commitment, travel distance and whether the trial will affect your personal or professional obligations.

Photos courtesy of Shutterstock

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SOURCE:
Crohn’s & Colitis Foundation


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