Dave Lambillotte’s Post

In January 2024, the Medicines and Healthcare products Regulatory Agency (MHRA) approved Valmorolone in the U.K. for treating Duchenne muscular dystrophy (DMD) in patients 4 and older. NICE are now in the process of reviewing it in order to make it available to patients on the NHS. But what do you know about it? What makes it different from the other steroid medications that are used as a treatment for Duchenne? Is it another steroid or a completely different medication? What side effects does it have? John Marrin, our Science Communication Coordinator, has written an overview which answers all of these questions and more. Knowledge is power. https://lnkd.in/eqwV3HeU #duchennescience #bitesizedscience #steroids #understanding #research #treatment #knowledge

Clinical Trials Day on May 20 ,in recognition of what is often considered the start of the first Randomized Clinical Trials. In 1947,James Lind began a Clinical Trial on this day to study the effects of different remedies for Scurvy among sailors at sea. Research provided evidence of the benefits of citrus fruits to treat this disease. Happy Clinical trials day for the individuals who participate in Clinical Trials as well as those professional who help run Clinical research. #Clinicaltrailsday #clinicaltrail #webinar #clinicalresearch #healthcare #who #contractresearchorganization #healthscience #lifescience #Pharmacy

💡 A “truly remarkable” form of #LSD has reportedly been granted ‘breakthrough therapy’ status by the US FDA after showing promise against Generalised Anxiety Disorder #GAD in phase 2b clinical trials. > The move comes after a single dose of the lysergide d-tartrate treatment - known as MM120 - achieved a “clinically and statistically significant” reduction in anxiety after a single oral dose. > According to biopharma company MindMed, which is developing the drug and conducted the trials, improvement in GAD symptoms was noted within two days of patients taking the drug. > The data – which also showed a 65% clinical response rate and 48% clinical remission rate after 12 weeks – persuaded the FDA to work closely with MindMed to assess MM120 in larger phase 3 trials. 🔬 Toronto Research Chemicals provides a wide range of chemical tools that support #DrugDiscovery and research into #MentalDisorders and #neurodegenerative diseases. Explore more at https://okt.to/iZryuQ #ScienceForASaferWorld

Only one day to register for the virtual event by International Society for Neonatal Screening! Register to hear from Erna Domsgen, PhD (CEO at ImmunoIVD AB) and other key opinion leaders from around the world discuss progress in programs, laboratory techniques and Ethical, Legal, and Social Implications (ELSI) aspects of #SMA screening. Find the registration link in the comments section below. #newbornscreening #spinalmuscularatrophy

Learn more about newborn screening for SMA!

💡 A “truly remarkable” form of #LSD has reportedly been granted ‘breakthrough therapy’ status by the US FDA after showing promise against Generalised Anxiety Disorder #GAD in phase 2b clinical trials. > The move comes after a single dose of the lysergide d-tartrate treatment - known as MM120 - achieved a “clinically and statistically significant” reduction in anxiety after a single oral dose. > According to biopharma company MindMed, which is developing the drug and conducted the trials, improvement in GAD symptoms was noted within two days of patients taking the drug. > The data – which also showed a 65% clinical response rate and 48% clinical remission rate after 12 weeks – persuaded the FDA to work closely with MindMed to assess MM120 in larger phase 3 trials. 🔬 Toronto Research Chemicals provides a wide range of chemical tools that support #DrugDiscovery and research into #MentalDisorders and #neurodegenerative diseases. Explore more at https://okt.to/4ZBrQC #ScienceForASaferWorld

Only one day to register for the virtual event by International Society for Neonatal Screening! Register to hear from Erna Domsgen, PhD (CEO at ImmunoIVD AB) and other key opinion leaders from around the world discuss progress in programs, laboratory techniques and Ethical, Legal, and Social Implications (ELSI) aspects of #SMA screening.    Find registration link in the comments section below. #newbornscreening #spinalmuscularatrophy

📰 Groundbreaking findings from LIFE-BTK now in the New England Journal of Medicine show: Esprit™ BTK drug-eluting resorbable scaffold demonstrated superior results vs angioplasty with respect to the primary efficacy endpoint among patients with #CLTI due to infrapopliteal disease. https://lnkd.in/gu7rAmZi Caution: Esprit™ BTK is an Investigational Device. Limited by Federal (USA) law for investigational use only. #ClinicalTrials #CLTI #PeripheralArteryDisease #Angioplasty

There's a common misconception that research participants are guinea pigs. But here's the truth: they're heroes! Consider this: Every medication you've ever taken has traversed a rigorous path through clinical trials. These trials, conducted over years, are meticulously designed not only to evaluate efficacy but also to prioritize patient safety. Throughout the process, data is continuously analyzed and reviewed to ensure that the highest standards of safety are upheld. In fact, safety is paramount at every stage of clinical trials. Before a new medicine even reaches human testing, it undergoes extensive laboratory research and preclinical studies to identify potential risks and ensure safety. Even during human trials, stringent protocols are in place to monitor and mitigate any adverse effects that may arise. Moreover, it's crucial to recognize that research participants are often the first to access new drugs for diseases. Their involvement in clinical trials provides them with early access to potentially life-saving treatments, offering hope where there may have been none before. So, next time you hear someone liken research participants to guinea pigs, remind them: they're the unsung heroes ensuring our medicines are safe, effective, and life-changing. #ClinicalTrials #MedicalAdvancements

Benzodiazepine Boom: Tracking Etizolam, Pyrazolam, & Flubromazepam from Pre-UK Psychoactive Act 2016 to Present Using Analytical & Social Listening Techniques - - Open Access Article from Pharmacy 2024, 12(1) >> https://loom.ly/Erdp3Fc