Amplity Health’s Post

The ongoing pursuit of addressing rare disorders necessitates a continuous drive for technological innovation, as current solutions often lack scalability and sustainability. Bridging these gaps through advancements in medical technology is crucial to establish a more effective and widespread approach to treating rare disorders. #raredisease #researchandinnovation

🎗️ In honor of Rare Disease Month, I'm committed to sharing news and facts on rare diseases throughout February. Kicking off with some pleasant good news: 📢 Health Canada has just approved MYALEPTA™ (metreleptin for injection) for patients with lipodystrophy (LD), a significant breakthrough for those living with this ultra-rare condition. 💜 As an adjunct to diet, MYALEPTA offers a new ray of hope, being the first treatment to address the complications of leptin deficiency in LD patients. This approval covers both congenital and acquired forms of generalized LD for adults and children aged two and above and familial or acquired partial LD for those aged 12 and above. 🔬 Brought to us by Chiesi Global Rare Diseases and developed by Amryt Pharma, MYALEPTA's availability in Canada is a major advancement for the LD community. It signifies a step forward in the treatment of this challenging condition, focusing on the underlying metabolic disorder to provide effective symptom control. 👏 The enthusiasm from medical professionals and patient advocates highlights the potential impact of MYALEPTA on enhancing the quality of life for individuals affected by lipodystrophy. This is a beacon of hope and a testament to the ongoing efforts in rare disease research and treatment development. 🎉As we continue to observe Rare Disease Month, let's celebrate this milestone and look forward to more innovations that promise a brighter future for patients battling rare conditions. ------------------------------------------------------------------------------------ Hello! I'm Sheena, a medical writer with expertise in #hematology #medicaldevices and #rarediseases . I partner with #medcomms , #cme firms, and companies in the #pharma and medical device sectors to create compelling narratives for various audiences. 📧 Connect with me to discuss your next project or follow along for insights into the world of a passionate #medicalwriter !

Pneumonia can present a wide array of symptoms, and understanding these is key to effective treatment. Our latest blog explores how Miami Clinical Research is uncovering new insights into pneumonia through targeted clinical trials—shedding light on overlooked symptoms and possible breakthroughs. Learn more about our journey in research here: https://lnkd.in/gDHRQEq6 #medicalresearch #clinicaltrials #medicalstudies #miamiclinicalresearch #pneumonia #pneumoniaresearch #lunghealth #pneumoniatreatment #pneumoniaclinicaltrials #respiratoryhealth

Serum GA is sensitive to diagnose IA in pediatric patients with excellent negative predictive value at an optimal cutoff of ≥0.7. Considering two consecutive values ≥0.7 increases specificity to 91.0%.

A real-world study assessed the use of maralixibat in children with Alagille syndrome who would not have been eligible for this treatment under clinical trial parameters and found that it effectively reduced pruritus from baseline. Read the full article here ⤵️ #AlagilleSyndrome #AlagilleSyndromeAlliance #CholestaticPruritus

There has been increasing interest to use clinical real-world data #RWD alongside clinical trial protocols #RCT in generating evidence in medical research #RWE e.g. to provide external control groups for single-arm studies. Data generation mechanisms, however, differ substantially in these two approaches. In our recent paper we make a rigorous evaluation of compatibility between datasets in patients with diabetic kidney disease and highlight the differences, but by machine learning we also demonstrate a partial overlap of RWD and RCT data. Jussi Leinonen, Samu Kurki https://lnkd.in/gQPS_-gm

To my fellow clinical research professionals, I want to use this medium to say thank you for your selfless sacrifices, dedication,passion and hardwork. You put in your time and energy into making historic events like this become a reality. Many of you go the extra miles to work after hours because you want to make sure clinical datas are correctly inputted into EDCs, you go extra miles to make sure participants are properly screened , you go extra miles to make sure sites are properly monitored , you go extra miles to make sure participants are properly followed up . The list is endless. Words are not enough to express my gratitude to the physicians that help monitor research participants. I want to also thank sponsors and CROs for giving birth to various clinical research initiatives which we all eventually run with. I want to say thank you . To the participants/Patients/Volunteers , we may not know your names , but we believe you know yourselves. We want to say thank you. You are the most important people in clinical research . I don’t like calling you our ‘patients’, I prefer the word ‘ participants’ because I believe you are the most important part of our clinical research team. On behalf of other clinical research professionals, I want to say thank you for volunteering to participate in various clinical trials . Some of you dint pass the eligibility checklists, some of you may have failed screenings, some of you may have successfully passed the screenings , some of you may have decided to withdraw during the trials, and some of you may have decided to stay to the end of the trial . You may belong to any of the above lists but for the fact that you even thought of participating in any clinical research trial, either you later changed your mind or decide to continue , you are automatically a special person to us. No clinical trial would be possible without you . Inshort you are the lubricant , the engine room, the lifeline of clinical research. What baffles me most, is the fact that some of you kept on with the trial even after we informed you of the risk and side effects and even added that you may not directly benefit from the trial. You still remained unshakened about your decision to partake in the trial. I want to say you are awesome . Centuries to come, the world of medicine will not stop celebrating you ,for taking the bold step to making our world a better place. To our future participants, we want to say thank you in advance . We love you all. Together we will make our world safer and healthier. See you at the top! Emmanuel Ndukwudimma https://lnkd.in/e6XjHzCh

Systemic lupus erythematosus (SLE) remains a rather mysterious disease that causes a multisystemic autoimmune disease characterised by the presence of autoantibodies towards nuclear antigens, immune complex deposition, and chronic inflammation at classic target organs such as skin, joints, and kidneys. End stage renal failure can follow in severe cases (mainly in women including young aged). Despite substantial advances in the diagnosis and management of SLE, the burden of disease remains high. It is important to appreciate the typical presentations and the diagnostic process to facilitate early referral and diagnosis for patients. Particularly in severe cases. In most patients, constitutional, mucocutaneous, and musculoskeletal symptoms represent the earliest complaints; these symptoms can include fatigue, lupus-specific rash, mouth ulcers, alopecia, joint pain, and myalgia. https://lnkd.in/dsJTKRvv End-stage renal disease and permanent haemodialysis are severe adverse events than can occur due to juvenile-onset and adult-onset systemic lupus erythematosus, with 15% of all patients with lupus nephritis developing end-stage renal disease. The therapeutic goal of complete remission of lupus nephritis is only reached in 60% of patients by conventional therapies, including immunosuppressive drugs and B-cell-depleting antibodies, and two-thirds of adults with juvenile-onset systemic lupus erythematosus develop organ damage and impaired health-related quality of life without reaching drug-free remission. Due to their high B-cell depletion activity, CD19-targeted chimeric antigen receptor (CAR) T cells are a potentially powerful strategy to treat autoimmune diseases, such as systemic lupus erythematosus. In a small case series of eight patients aged 18–38 years with treatment-refractory systemic lupus erythematosus, adoptive transfer of CD19-targeted CAR T cells induced a deep reset of B cells leading to abrogation of autoreactive antibodies and durable remission, including abrogation of lupus nephritis with a follow-up time of 6–29 months. Now a specialized German group report positive results of CAR T therapy on a 15-year-old female patient with severe and rapidly progressive systemic lupus erythematosus. Worth of a larger prospective and randomized clinical trial as compared to standard therapy and supportive treatment? #CART #SLE #CD19 #autoimmune

Thrombophilia is one of the multiple inherited or acquired risk factors to thrombosis. Did you know that in conducting thrombophilia screening, it is important to thoughtfully evaluate the potential clinical benefits for both the patient and their family members? Watch our video on Thrombophilia here: https://ow.ly/4cWv50RiOIR #Meded

How do you identify and manage your patients with diabetic kidney disease? Professor Tina Vilsbøll provides an overview of the diabetes-cardio-kidney spectrum and the importance of proactive management of diabetic kidney disease (DKD) in our NEW expert-led program "T2D and CKD: putting evidence into practice". This program will feature discussion vodcasts with updates from leading experts in the field and patient case studies to allow you put your learnings into practice. Be sure to register to the program to recieve updates when new content is published. Available at: https://lnkd.in/eJD4r7Jx --- The information and data provided in this program was updated and correct at the time of the program development, but may be subject to change. This educational activity is intended for an international audience of non-US and non-UK HCPs. Supported by an educational grant from NOVO NORDISK A/S. #CKD #T2D #type2diabetes #chronickidneydisease #nephrology #diabetes #diabetesmanagement #medicaleducation #continuingmedicaleducation #cme #independentmedicaleducation #ime #SpringerIME