FDA CDC News
FDA Approval of Nonsteroidal Treatment for Duchenne Muscular Dystrophy
Breaking news! FDA approves Duvyzat for Duchenne Muscular Dystrophy, a nonsteroidal treatment providing hope for patients and families. #DMD #FDAApproval
Breaking news in the medical world! The U.S. Food and Drug Administration has just approved Duvyzat (givinostat), an oral medication for the treatment of Duchenne Muscular Dystrophy (DMD) in patients six years of age and older. Duvyzat is making history as the first nonsteroidal drug approved to treat patients with all genetic variants of DMD. This is a significant milestone in the treatment of this devastating disease.
DMD, being the most common childhood form of muscular dystrophy, primarily affects males. It is a neurological disorder that leads to progressive muscle weakness due to a lack of dystrophin, a muscle protein. Over time, the muscles deteriorate, causing difficulties with walking, muscle strength, and eventually leading to breathing problems and early death. However, with advancements in treatment, the life expectancy for individuals with DMD has been steadily increasing, with some patients surviving beyond 30 years.
Duvyzat is a histone deacetylase (HDAC) inhibitor that targets pathogenic processes to reduce inflammation and loss of muscle in patients with DMD. Its efficacy for the treatment of DMD was evaluated in an 18-month phase 3 study involving a randomized, double-blind, placebo-controlled trial. The primary endpoint of the study was the change in muscle function, measured by the time it took patients to climb four stairs. Patients treated with Duvyzat showed a statistically significant reduction in the decline of muscle function compared to placebo.
Another measure of efficacy was the change in physical function assessed by the North Star Ambulatory Assessment (NSAA), a scale commonly used to rate motor function in boys with DMD who can still walk. Patients treated with Duvyzat experienced less worsening in their NSAA scores after 18 months, compared to those on placebo.
Like any medication, Duvyzat does come with some potential side effects. The most common ones reported were diarrhea, abdominal pain, a decrease in platelets, nausea/vomiting, an increase in triglycerides, and fever. It’s important for healthcare providers to evaluate a patient’s platelet counts and triglyceride levels before prescribing Duvyzat. Patients with low platelet counts should not take the drug. Monitoring of platelet counts and triglycerides throughout treatment will help determine if any dosage adjustments are needed.
Healthcare professionals should also be aware that Duvyzat may cause QTc prolongation, which can increase the risk of irregular heartbeats. It’s crucial for patients taking other medications known to cause QTc prolongation or with certain types of heart disease to avoid taking Duvyzat.
The recommended dosage of Duvyzat is determined by the patient’s body weight, and it should be taken orally twice daily with food.
The approval of Duvyzat was granted to Italfarmaco S.p.A., and it received priority review, fast-track designation, as well as orphan drug and rare pediatric disease designations from the FDA. This demonstrates the agency’s commitment to advancing the development of new therapies for DMD and its recognition of the urgent need for effective treatments.
This milestone approval represents hope and progress for individuals and families affected by DMD. It provides another treatment option to help reduce the burden of this progressive and debilitating disease, regardless of the specific genetic mutation. With continued advancements in medical research and the dedication of organizations like the FDA, we are moving closer to a future where individuals with DMD can lead healthier lives.
As always, it’s important to consult with healthcare professionals for more information and guidance regarding Duvyzat and its suitability for individual cases. Let’s celebrate this achievement in medical science and look forward to more groundbreaking developments in the treatment of Duchenne Muscular Dystrophy.
Source: FDA
Related Information
- Duchenne muscular dystrophy – About the Disease – Genetic and Rare Diseases Information Center
- The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.
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FDA CDC News
Seafood Recall: Important Information on Hannaford Seafood Salads
In early November, the U.S. Food and Drug Administration (FDA) announced a Class II recall concerning Hannaford Seafood Salads, affecting over 1,600 units across five states: Maine, New Hampshire, Vermont, Massachusetts, and New York. The recall, initiated on October 31, was due to a mislabeling incident that poses a significant risk to consumers with soy allergies.
Understanding the Class II Risk Level
The FDA has classified this recall as a Class II risk, indicating that exposure to the affected products may cause “temporary or medically reversible adverse health consequences.” However, the risk of experiencing serious health consequences remains “remote.” Despite this classification, consumers with an allergy or severe sensitivity to soy must take this recall seriously, as they could face life-threatening allergic reactions if they consume the mislabeled salads.
What Happened?
The problem arose when the printer responsible for the salad labels provided Elevation Foods, the manufacturer of the salads, with labels that did not accurately reflect the current product formulation. Instead of the correct labels, the printer mistakenly issued labels meant for an upcoming formulation change. As a result, the affected salads may contain undeclared hydrolyzed soy protein, a known allergen that must be clearly stated on product labels to protect consumer safety.
Product Details
The implicated Hannaford Seafood Salads were distributed between October 25 and 29, and they feature use-by dates of November 13 and 14. Consumers can find these dates printed on the lid of the containers. Following the recall announcement, all affected products were promptly removed from store shelves.
Health Risks Associated with Soy Allergies
For individuals with soy allergies, the immune system mistakenly identifies soy proteins as harmful invaders, leading to a range of symptoms that can vary in severity. Common reactions include stomach issues, coughing, itching, wheezing, vomiting, hives, and difficulty breathing. In severe cases, these reactions can escalate to life-threatening conditions, such as anaphylaxis.
To mitigate risks, those with soy allergies are advised to carry an epinephrine injection (Epi-Pen) to counteract severe allergic reactions swiftly.
What Should Consumers Do?
Customers who purchased Hannaford Seafood Salads before the recall are urged not to consume the products. Instead, they can return them to the store where they made the purchase for a full refund. Elevation Foods has not reported any known cases of illness related to these salads, but consumer safety remains a top priority.
Conclusion
This seafood salad recall serves as a crucial reminder about the importance of accurate labeling, especially when it comes to potential allergens. Consumers should remain vigilant and informed about the products they purchase, particularly if they have food allergies. Stay safe and always check your labels!
Related Links:
https://www.newsweek.com/seafood-salad-recall-update-fda-risk-level-hannaford-1992294
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pets
Urgent Dog Treat Recall: Carolina Prime Pet Issues Warning Due to Salmonella Risk
Dog Treat Recall
In a recent announcement that has concerned dog owners across the Southeastern, Midwestern, and Mid-Atlantic regions of the United States, the FDA has reported a recall of “Hollywood Feed Carolina Made Chicken Chips” dog treats manufactured by Carolina Prime Pet, Inc. of Lenoir, North Carolina. This recall, affecting approximately 400 bags of these popular dog treats, is attributed to the potential risk of Salmonella contamination.
Why Is This Recall Important?
While there have been no reported illnesses linked to the consumption of these chicken chips, the safety of pets—and by extension, their human companions—is paramount. Salmonella, a bacteria that can cause severe gastrointestinal distress in animals and humans alike, can be particularly dangerous for pets, leading to symptoms such as vomiting, diarrhea, and lethargy. For individuals, Salmonella can also result in serious health complications, especially in young children, elderly adults, or those with weakened immune systems.
Details of the Recall
The recall specifically pertains to 16-ounce bags of “Hollywood Feed Carolina Made Chicken Chips” that carry the lot number 20051324L2 and an expiration date of 11/13/25. Consumers can easily identify the affected product by checking the back of the packaging, which features a distinctive purple-and-black design. The recall extends to retail locations across 19 states, including:
- Alabama
- Arkansas
- Florida
- Georgia
- Illinois
- Indiana
- Kansas
- Kentucky
- Louisiana
- Michigan
- Mississippi
- Missouri
- North Carolina
- Ohio
- Oklahoma
- Pennsylvania
- South Carolina
- Tennessee
- Texas
For those who purchased the treats online, they were also available on the company’s website at www.hollywoodfeed.com.
What Should Consumers Do?
Carolina Prime Pet, Inc. has advised consumers to check their homes for the affected product. If your dog treats match the recalled lot number and expiration date, it is crucial not to feed them to your pet. Instead, return the products to the place of purchase for a full refund. For those who have questions or need more information, the company encourages contacting their customer service.
Final Thoughts
As pet owners, it is our responsibility to stay informed about the products we give our furry friends. The safety of our pets is essential, and recalls like these serve as a reminder to always check product packaging and stay updated on food safety announcements. If you have any concerns about your pet’s health or have noticed any unusual symptoms after consuming potentially contaminated food, it is advisable to consult with a veterinarian immediately.
Stay alert, stay informed, and together we can ensure the safety and well-being of our beloved pets.
Related links:
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FDA CDC News
Popular Nasal Decongestant Falls Short: FDA Advisers Question Efficacy
FDA questions effectiveness of popular nasal decongestant phenylephrine, raising concerns about congestion relief.
In a surprising turn of events, government experts have cast doubt on the effectiveness of the leading decongestant found in popular over-the-counter medications like Sudafed and Dayquil. A recent review conducted by the Food and Drug Administration (FDA) found that the main drug ingredient, phenylephrine, offers no significant relief from nasal congestion. This revelation raises concerns among millions of Americans who rely on these medications for congestion relief.
Phenylephrine became the primary ingredient in over-the-counter decongestants after pseudoephedrine, an older ingredient, was moved behind pharmacy counters due to its potential for illegal drug processing. However, the FDA’s outside advisers unanimously voted against the efficacy of phenylephrine, prompting a reevaluation of its effectiveness. Despite phenylephrine-based versions dominating the market, accounting for four-fifths of the $2.2 billion oral decongestant market, the FDA’s conclusions suggest a need to reconsider their availability.
The FDA review identified significant flaws in the original studies that supported phenylephrine’s approval. These studies, conducted in the 1960s and 1970s, were deemed inadequate by modern standards. Furthermore, three recent, well-conducted studies by reputable pharmaceutical companies found no discernible difference between phenylephrine medications and placebos in relieving congestion. This raises concerns about the effectiveness of phenylephrine, which has relied on outdated research.
The FDA’s advisory panel’s unanimous vote against phenylephrine’s effectiveness could lead to regulatory action requiring drugmakers like Johnson & Johnson and Bayer to withdraw their phenylephrine-based oral decongestants from store shelves. This move may force consumers to opt for behind-the-counter pseudoephedrine products or phenylephrine-based nasal sprays and drops. However, it would necessitate extensive education efforts by the FDA, pharmacists, and drugstores to inform consumers about alternative options for treating congestion.
The Consumer Healthcare Products Association, representing nonprescription drugmakers, argued that the recent studies have limitations and advocated for continued “easy access” to phenylephrine. However, the FDA review highlighted the shortcomings of the original studies and the lack of modern scientific evidence supporting phenylephrine’s efficacy. Balancing public health concerns and consumer access will be a crucial challenge for regulators moving forward.
The FDA’s recent review has cast doubt on the effectiveness of phenylephrine, the key ingredient in popular nasal decongestants. This finding prompts a reevaluation of treatment options for nasal congestion and calls for further research to provide effective relief for individuals in need.
Check out the article from the Associated Press: https://apnews.com/article/sudafed-decongestants-phenylephrine-pseudoephedrine-fda-0f140bafae9a500c5fba05fe764ecb66
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