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Single drug provides first evidence of 'nearly universal' pharmacological chaperone for rare disease

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  • 2025-09-22 16:00 event
  • 2 hours ago schedule
Single drug provides first evidence of 'nearly universal' pharmacological chaperone for rare disease
A study published in Nature Structural & Molecular Biology is the first time researchers have shown evidence that a single drug, already licensed for medical use, can stabilize nearly all mutated versions of a human protein, regardless of where the mutation is in the sequence.

6.607. This Is The Unique Sunscreen Pam And Hailey Have Been Using

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According to Instagram, Bieber counts the multitasking formula among her empties.View Entire Post ›

6.608. Drew Barrymore Is Being Called “Real And Genuine” After Documenting Her “First Perimenopause Hot Flash” On Live TV While Interviewing Jennifer Aniston And Adam Sandler

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“I don’t know that I have ever heard a celebrity talk about a hot flash in the moment. Thank you for being so real.”View Entire Post ›

6.609. This $16 French Moisturizer Is Amazon’s Best-Kept Beauty Secret

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The Embryolisse Lait-Crème Concentré is a multi-tasking hidden gem of a facial cream.View Entire Post ›

6.610. Lila Moss Opened Up About What It Was Like Being Diagnosed With Type 1 Diabetes

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After the model opened up about her experience being diagnosed with type 1 diabetes, we asked experts about the autoimmune condition, the differences between type 1 and type 2 diabetes, and how this diagnosis can change your life.View Entire Post ›

6.611. Why Reviewers Swear By This $28 Tool For Back Pain Relief

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An acupuncturist explains how this scary-looking acupressure mat can help relieve back pain.View Entire Post ›

1. Activity of large-scale cortical networks follows cyclical pattern, study finds

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The human brain can concurrently support a wide range of advanced mental functions, including attention, memory and the processing of sensory stimuli. While past neuroscience studies have gathered valuable insight into the neural underpinnings of each of these processes, the mechanisms that ensure that they are performed efficiently and in a timely fashion have not yet been fully elucidated.

2. Study shows UV light can disable airborne allergens within 30 minutes

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Cats, dust mites, mold, trees; for people with allergies, even a brief whiff of the airborne allergens these organisms produce can lead to swollen eyes, itchy skin and impaired breathing.

3. Psychological distress common after a heart attack, may lead to future heart conditions

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Many people experience one or more types of psychological distress, including depression, anxiety, psychosocial stress or post-traumatic stress disorder (PTSD), after a heart attack, which can affect their physical recovery and long-term heart health, according to a new American Heart Association scientific statement, published in Circulation.

4. Our actions are dictated by 'autopilot' not choice, finds study

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Habit, not conscious choice, drives most of our actions, according to new research from the University of Surrey, University of South Carolina and Central Queensland University.

5. Single drug provides first evidence of 'nearly universal' pharmacological chaperone for rare disease

  • 2 hours ago schedule
  • medicalxpress.com language

A study published in Nature Structural & Molecular Biology is the first time researchers have shown evidence that a single drug, already licensed for medical use, can stabilize nearly all mutated versions of a human protein, regardless of where the mutation is in the sequence.

6. Study reports on recent trends in opioid prescribing for patients with cancer

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A recent analysis reveals a modest decline from 2016 to 2020 in new and additional opioid prescriptions for patients with cancer. Among those patients with metastatic cancer, prescribing remained stable for those reporting any pain and declined steeply for those reporting no pain. The findings are published in the journal Cancer.

7. Study finds virtual care expansion did not expand specialist access in rural areas of Canada during the pandemic

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Despite the expansion of virtual care in Ontario prompted by the COVID-19 pandemic, specialist physicians did not expand their reach to patients living at great distances from where they provided care, finds research published in the Canadian Medical Association Journal.

8. Scientists call for urgent action to reduce children's plastic exposure

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Childhood exposure to chemicals used to make plastic household items presents growing health risks that can extend long into adulthood, experts from NYU Langone Health report.

9. Concussion, identity loss, depression: Boxing's toughest opponent isn't in the ring—it's mental health

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Ricky Hatton's death has reignited an all-too-familiar conversation about mental health in sport. Hatton had spoken openly about his long battle with depression, as well as the drug and alcohol addiction that began after his 2007 defeat to Floyd Mayweather.

10. Long COVID associated with abnormal uterine bleeding and cycle-phase symptom variation

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The Center for Reproductive Health, University of Edinburgh reports that long COVID was associated with abnormal uterine bleeding in a UK population, with increased menstrual volume, longer duration and more intermenstrual bleeding with no impaired ovarian function, alongside differences in peripheral and endometrial inflammation.

11. Why mosquitos might be attracted to you

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Researchers in the Netherlands turned a major music festival into an unexpected laboratory to investigate a question that has long puzzled scientists and bug-bitten individuals alike: What makes some people more irresistible to mosquitoes than others?

12. Does ASMR really help with anxiety? A psychology expert explains the evidence

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Most of us have experienced tingling or "goosebumps" at some point, especially when we feel a strong positive emotion such as awe or excitement.

13. Is it OK to sit on public toilet seats?

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If you're a parent or have a chronic health condition that needs quick or frequent trips to the bathroom, you've probably mapped out the half-decent public toilets in your area.

14. How much progress has been made against Alzheimer's disease?

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After decades of unsuccessful research, two new drugs and a pioneering blood test have recently given Alzheimer's patients hope of fighting back against the debilitating disease—but questions remain about their effectiveness.

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