Mineralys Therapeutics, Inc.’s Post

For patients living with Prader-Willi Syndrome (PWS), a life threatening, life-shortening rare genetic disease, there is no time to lose. Left untreated, patients suffer from physical, mental, and behavioral challenges that can lead to death- and there’s currently no treatment on the market to address the characteristic insatiable hunger. Soleno Therapeutics, Inc. is bringing new hope to the PWS community, but the company has lost three years to regulatory delays and obstacles. Every day is critical to patients living with PWS, who have an average lifespan of 21-29 years - and a three-year delay can mean the difference between life and death for patients. “The impact of regulatory inconsistency, delays, and lack of clarity has a disproportionate impact on rare disease communities – 95% of which have no FDA-approved treatment. For every day that goes by, symptoms will worsen, sometimes in an irreversible manner, and patients may even die as they wait for treatments.” Anish Bhatnagar, CEO of Soleno Therapeutics knows this reality well, and the RDCC agrees. There must be a balance between rigorous regulatory review and urgency to deliver treatments to patients that are desperately seeking hope. #OneRareVoice #LifeScience #Healthcare #RareDiseaseWeek

A drug delivery vehicle for treatment of kidney injury: Fibroblastic reticular cells- Exosomes (FRC-Exos) • Researchers investigated (linked below), the therapeutic effect of Fibroblastic reticular cells (FRCs) derived exosomes in sepsis-induced acute kidney injury using a mouse model. They have demonstrated that FRC-Exos improved kidney function, survival rate, and mitochondrial integrity in the kidney. • FRC-Exos, which are rich in CD5L, promoted PINK-PARKIN-mediated mitophagy in primary renal epithelial cells, resulting in the degradation of the NLRP3 inflammasome and improved kidney function. CD5L is an immunoregulatory protein. • Furthermore, the modification of FRC-Exos with LTH, a targeting peptide that targets KIM-1, allowed FRC-Exos to selectively adhere to kidney tubular cells thereby enhancing the therapeutic efficacy of Exosomes-based treatment. #murine model of sepsis-induced acute kidney injury #chronickidneydisease # Fibroblasticreticularcells #exosomes #mitophagy #kidneystory #scientificcommunications #preclinicalresearch Fibroblastic reticular cell-derived exosomes are a promising therapeutic approach for septic acute kidney injury - Kidney International (kidney-international.org)

Immunic Therapeutics (NASDAQ:IMUX) president and CEO Dr Daniel Vitt joined Thomas Warner from Proactive ahead of World Brain Day to provide an update on the #biotechnology company's novel #multiplesclerosis (MS) treatment. Dr Vitt gives an overview of the treatment that #Immunic is currently working on, and emphasised the significance of addressing the high unmet medical need for #MS patients worldwide. Immunic's focus is on developing a new therapeutic option, using vidofludimus calcium, to treat both relapsing and progressive forms of MS. He days that the drug is the first known modulator of the "so-called Nurr1 target", making it a potential neuroprotective treatment. Immunic is conducting a phase two clinical study into its treatment for progressive MS, with biomarker data expected in the second half of the year. Watch at #Proactive #ProactiveInvestors #NASDAQ #IMUX https://ow.ly/wFlI50Pi6Mr

Successful drugs...extend life among other things. There can be a soft underbelly to these success stories. This Boston Globe article on Vertex's cystic fibrosis medications sensitively discussing how the medications change CF patients' lives in both positive and (surprisingly) negative ways. #cysticfibrosis #lifechangingmedications #qualityoflife https://lnkd.in/ejUzyWGU

📚 Today, let's immerse ourselves in the world of knowledge with "The LDN Book 3" – an invaluable resource delving into the realm of Low Dose Naltrexone (LDN). 📖🔍 Inside these pages lies the latest research, uncovering LDN's impact on viral infections, mold toxicity, longevity, cancer, depression, and more. It's a testament to the ever-evolving landscape of health and wellness solutions. For a deeper understanding, follow the link below to explore LDN's transformative potential and its far-reaching implications in our professional practice. 👉 Explore Further:

Join the Anovo team as we observe World Homocystinuria Awareness Day, standing in solidarity with those affected by this rare metabolic disorder. Homocystinuria (HCU) is a rare genetic condition that affects approximately 1 in 200,000 individuals worldwide. It's caused by the body's inability to process the amino acid methionine, leading to the accumulation of harmful substances in the blood and urine. This can result in serious health issues such as heart disease, vision problems, and intellectual disabilities if left untreated.   Anovo is proud to support patients and families who rely on specialty and orphan drugs to treat HCU. Our partnership with Recordati Rare Diseases North America enables us to bring a promising treatment to market that helps reduce homocysteine levels in the blood, improving the lives of those with this rare condition. Let’s shine a light on World Homocystinuria Awareness Day and show our commitment to making a difference in the lives of HCU patients and their caregivers. #HomocystinuriaAwareness #RareDisease

Urgent Support Needed: Uniting for Research on Post-Finasteride Syndrome Today, I am compelled to address an urgent cause, Post-Finasteride Syndrome (PFS) is a grave and devastating condition resulting from the use of the drug Finasteride (marketed as Propecia or Proscar). Sadly, someone close to me is suffering from PFS, and currently, there are neither cures nor adequate support available for those affected. Time is a luxury we cannot afford. Ongoing research efforts at institutions such as Bringham and Women's Hospital and Baylor College of Medicine desperately need financial support. Merck Group Merck Life Science, the manufacturer of Propecia, has an ethical responsibility to actively participate in this research. Your Signature can Make a Difference: - Accountability: It’s time for Merck to face the repercussions of their product. - Urgency: The lives of those suffering cannot wait. - Solidarity: Let’s show that we care and are ready to act. By signing and sharing, we can support a vital cause. Your action can catalyze the necessary change. Thank you for your support and solidarity. https://lnkd.in/eesGTk4r

Hey guys, November is all about you! It’s Men’s Health Month! Prostate cancer, mental health, testicular cancer, and suicide prevention are all important and real issues for New Zealand men. Movember is a great opportunity to raise awareness of these issues. If you or someone you know is affected, reach out and get some support. The Movember website is jam packed full of advice and tips on how to be more aware of the issues that may affect your health and how you can get support: https://nz.movember.com #medicine #healthcare #pharmaceuticals #pharma #movember #menshealth #prostatecancer #clinicalresearch #mentalhealth #testicularcancer #suicideprevention

The journey to know more about lipedema. 👉 We know that when we feel alone in a situation like this, it can be hard to believe that other people are going through it, too. But the truth is that even if you've never heard of lipedema before today, chances are good that someone you know has had it. In fact, according to the National Lipedema Association (NLA), up to 2 million Americans may have lipedema (though some experts believe this number may be even higher). So, if you're curious about lipedema—or if you think someone close to you might have it—take a moment to learn more about this condition and how to support your loved ones. 📌 Take the Lipedema Quiz to learn more about lipedema. ➡ https://lnkd.in/encMpmPx

Behind every patient there is a story. Behind every story there is a human being. In my journey having worked in rare diseases for the last 17 years, I've learned that behind each patient, there's always a riveting story. A story of resilience, of strength, of battles fought and sometimes lost. A story that shapes their health journey and influences their response to treatment. Take John. He was a 55-year-old patient who battled both small lung cell cancer and the Lambert Eaton-Syndrom, and this during the isolation of the pandemic - physically apart from loved ones yet holding a steadfast courage in his heart. His story is a testament to the resilience of the human spirit. And then there's Claudine. A single mother juggling two jobs and facing the verdict of her 2 sibling-twins diagnosed with a genetic lysosomal storage disease. The strength she demonstrates as she balances her health, her work, and her family's welfare is nothing but awe-inspiring. Behind every medical chart, there's a life filled with emotions, experiences and dreams. That's why as a pharmaceutical professional, it is vital that we not only raise awareness across rare diseases, but also that we understand, empathize, and connect. Recognizing these stories refines our approach to care and encourages a healthier patient-doctor relationship. Medicine is not just science, but it is also an art of understanding the human narrative. #empathy #rarediseaseday #raredisease #orphandrugs #medical #pharma #biotech #pharmeceuticals #hope #storytelling #listen