Alaa Hamed, MD, MPH, MBA’s Post

📢 Important update on our PMD-OPTION study in Primary Mitochondrial Disease #PMD! ⬇ We have completed enrollment of our Phase 2 PMD-OPTION clinical study in PMD and are on track to have top-line data available mid-2025. Initial data analysis confirms the strong safety profile and very good tolerability of OMT-28 in the target population. Furthermore, the clinical data obtained so far looks promising for relevant endpoints, which bodes well for our goal to provide a much-needed new treatment option for patients underserved by current therapies. Want to learn more? Meet OMEICOS during the upcoming #BIOEurope Partnering Conference in #Stockholm, November 4-6th. #mitochondrialdisease #mitoawareness #mitochondrialresearch

Good news from @omeicos on the currently running clinical phase 2 trial in rare disease indication Primary Mitochondrial Disease: - enrollment in PMD-OPTION study completed - enrollment target met - strong safety and tolerability of OMT-28 shown and - on track for top-line results next year. Great commitment by patients for our study and big achievement of the whole PMD-OPTION study teams at the clinical sites, the CROs and at OMEICOS #teamwork #mitochondrialdisease #clinicalstudy

🔴 NEW report launch: Delivering Effective Treatments for Rare Disease Patients Rare diseases impact millions, yet developing and delivering effective treatments remains a significant challenge. Public Policy Projects' latest report highlights critical obstacles and innovative opportunities to improve treatment access for those living with rare and ultra-rare conditions. Key insights: 👉 Patient Engagement Matters: Building trust through clinical geneticists and involving patients in trial design can drive awareness and participation. 👉 Registries Are Critical: Patient support organisations play a vital role in creating condition-specific registries, bridging gaps in clinical trial participation. 👉 Opportunities for Innovation: Drug repurposing and NHS workforce training in rare disease research offer exciting avenues to advance treatment delivery. This report calls on industry, government, the NHS, and all stakeholders to take bold action. As Co-Chair Alastair Kent OBE states, it’s time to “deliver innovative interventions that will effectively address these unmet needs.” 📚 Access the full report here: https://lnkd.in/dCWs_BRV 🤝Thank you to our contributors, partners, and sponsors who made this possible. Sponsors: Costello Medical, Fortrea, Amicus Therapeutics and Bayer UK. Partners: Genomics England, Wellcome Sanger Institute and NIHR BioResource. #RareDiseases #PatientEngagement #ClinicalTrials #HealthInnovation

Proud to support the management of #myastheniagravis in Belgium! Myasthenia gravis cannot be cured, but with proper and patient-tailored disease management many people can live their normal lives again, with minimal to no symptoms. Clinical guidelines can strongly improve physicians in providing the best possible care for patients.    As #optimisinghealthcare is what #Hict'ers live for we are proud to announce the publication of #clinicalrecommendations for the management of myasthenia gravis in Belgium: ✔ based on the latest scientific evidence; ✔ aligned with the Belgium reimbursement criteria; ✔ keeping in mind newer and (near) future therapies; ✔ with a focus on the practical implementation to optimally support our Belgian neurologists.   The manuscript has been published in #ActaNeurologicaBelgica. Read it here! https://lnkd.in/eCxN88G8    We truly appreciated collaborating with the authors, experts from the seven Belgian neuromuscular reference centers PROF. DR DE BLEECKER JAN, Kristl Claeys, Stéphanie Delstanche, Bissay, Véronique, Vinciane Van Parijs, Gauthier Remiche, Alicia Alonso-Jiménez and want to thank Alexion Pharmaceuticals, Inc., argenx, Johnson & Johnson, UCB for their support. Want to know more on how Hict handles such projects? Read the reference case on our website: https://lnkd.in/eiPdS8Mk

Rare disease trials are complex due to small, diverse patient populations and intricate trial designs. Gain critical insights from experts in #raredisease #clinicaltrial design in our new e-book, which reveals common issues, solutions and the expertise required to enhance success. Learn how to navigate barriers and implement effective strategies for rare disease #drugdevelopment, and empower your team with the knowledge to innovate and bring life-saving therapies to market.

🧬 The Primary Challenge in Rare Disease Clinical Trials—And a Ready Solution! 🧬 • 🌍 7,000+ rare diseases globally, but only a fraction have available therapies. • 🔍 Patient recruitment for clinical trials remains a significant challenge. • 🌐 Expanding to global sites in regions like USA and APAC can unlock untapped patient populations. • 🚑 Biopharma companies are targeting new regions to recruit rare disease patients. 💡 At Credevo, we’ve successfully helped find patients for rare conditions like Glomerulonephritis, ALS, and more. 🚀 Let's explore how we can accelerate your trial development! 🔗 Discover more with Credevo! https://lnkd.in/gJBT6iZk #RareDiseases #ClinicalTrials #Credevo

🧬 The Primary Challenge in Rare Disease Clinical Trials—And a Ready Solution! 🧬 • 🌍 7,000+ rare diseases globally, but only a fraction have available therapies. • 🔍 Patient recruitment for clinical trials remains a significant challenge. • 🌐 Expanding to global sites in regions like USA and APAC can unlock untapped patient populations. • 🚑 Biopharma companies are targeting new regions to recruit rare disease patients. 💡 At Credevo, we’ve successfully helped find patients for rare conditions like Glomerulonephritis, ALS, and more. 🚀 Let's explore how we can accelerate your trial development! 🔗 Discover more with Credevo! https://lnkd.in/gXTSzFgZ #RareDiseases #ClinicalTrials #Credevo

New data to help in treatment decisions for Crohn's disease patients! A study recently published in the New England Journal of Medicine shows that risankizumab, brand name SKYRIZI® is more effective than ustekinumab, brand name STELARA® in achieving clinical and endoscopic remission in patients with moderate-to-severe Crohn’s disease who had unacceptable side effects with anti-TNF therapy or an inadequate response to such therapy. Significant strides in treatment options! Find the clinical evidence for both #SKYRIZI & #STELARA on DRUGDOCS® Read the paper here: https://lnkd.in/ex5znVzp #CrohnsDisease #MedicalResearch #IBD

🧬 The Primary Challenge in Rare Disease Clinical Trials—And a Ready Solution! 🧬 • 🌍 7,000+ rare diseases globally, but only a fraction have available therapies. • 🔍 Patient recruitment for clinical trials remains a significant challenge. • 🌐 Expanding to global sites in regions like USA and APAC can unlock untapped patient populations. • 🚑 Biopharma companies are targeting new regions to recruit rare disease patients. 💡 At Credevo, we’ve successfully helped find patients for rare conditions like Glomerulonephritis, ALS, and more. 🚀 Let's explore how we can accelerate your trial development! 🔗 Discover more with Credevo! https://lnkd.in/g_vqqFid #RareDiseases #ClinicalTrials #Credevo

A patient-led organization has just made an incredible breakthrough by repurposing a drug for a rare and deadly bleeding condition! Recently, a large clinical trial revealed that pomalidomide, originally developed for multiple myeloma, has proven effective in treating a life-threatening bleeding disorder called HHT (Hereditary Hemorrhagic Telangiectasia). This drug not only reduced bleeding events but also improved patients’ quality of life. Cure HHT brought together physicians, researchers, and patients to push this repurposing opportunity forward, showing the true power of patient-led organizations. At Every Cure, we aim to work closely with patient organizations to identify the most promising opportunities from our platform and ensure the necessary trials are conducted to benefit as many patients as possible. Because no one understands a rare disease better than those living with it. #raredisease #drugdevelopment #drugrepurposing #clinicaltrial