Rachael Fleurence, Ph.D.’s Post

🔬 👩🔬 It's International Clinical Trials Day! Last November, the final results of the MSF-led TB-PRACTECAL clinical trial were published. This trial identified a new shorter treatment regimen for drug-resistant tuberculosis (DR TB). The results of this trial led to the WHO recommending a 6-month all oral treatment for DR TB, which has been adopted for use in 40 countries until now. Last month (April 2024), MSF presented the costs of the TB-PRACTECAL clinical trial (€33.9 million). This is the first time the detailed costs of an individual clinical trial will have ever been shared publicly. Read more about why MSF thinks that increasing transparency in biomedical R&D costs would help shape more equitable models of R&D and ensure global access to lifesaving treatments for people who urgently need them: https://lnkd.in/ecU86Jwn #ICTD2024

February 29th marks Rare Disease Day, bringing awareness to conditions like CTCL, a rare form of T-cell NHL. Learn more about SciTech's ST-001, which holds an orphan drug designation. Orphan diseases, affecting fewer than 200,000 people in the US, pose serious or life-threatening risks. The Orphan Drug Act aims to foster research and develop drugs for these conditions. Learn more about rare diseases here https://lnkd.in/gk6hbAF and ST-001 here https://scitechsdp.com/. #RareDiseaseDay #CTCL #ST001 #OrphanDrugDesignation #scitechSDP #scitechdevelopment #nanofenretinide #fenretinide #lymphoma #nonhodgkin #ST001

𝗔𝘁 𝗮𝗻𝘆 𝗴𝗶𝘃𝗲𝗻 𝘁𝗶𝗺𝗲, 𝟰% 𝗼𝗳 𝘁𝗵𝗲 𝗴𝗹𝗼𝗯𝗮𝗹 𝗽𝗼𝗽𝘂𝗹𝗮𝘁𝗶𝗼𝗻 𝗴𝗿𝗮𝗽𝗽𝗹𝗲𝘀 𝘄𝗶𝘁𝗵 𝗮 𝗿𝗮𝗿𝗲 𝗱𝗶𝘀𝗲𝗮𝘀𝗲. Yet, of the estimated 10,000 rare diseases that have been identified, a staggering 95% lack FDA-approved treatments, leaving a vast majority searching for cures. These numbers underscore the intricate web of challenges in drug development for rare diseases—from diagnosis hurdles to regulatory pathways and the quest for sustainable treatments. Today, at the 19th Annual HD Therapeutics Conference, hosted by the CHDI Foundation, Alessandro Piai highlighted IRBM’s integrated approach and how we leverage our experience to drive even more progress toward the discovery of new therapeutics for rare diseases. 🧬 On this @Rare Disease Day, let's all recognize those who are affected by rare diseases and recommit to transforming the landscape of drug development, treatment, and care. Michele Luche Sara Tomaselli Carlo Toniatti Manuel Daldin Cristina Cariulo #rarediseaseday #huntingtonsdisease #irbm #hopeforacure

This is an example of what we do best with WHO guidelines and operational research. In this case by providing #TB doctors with the evidence, training and support needed to update clinical practice and give patients the best chance of recovery from hard-to-treat drug resistant forms of TB. Dr Liga Kuksa summed it up: “We are trained to practice evidence-based medicine and, thanks to this research, clinicians now feel comfortable using these shorter treatment regimens because we have both the evidence and the experience of seeing more people recover from MDR-TB.” https://bit.ly/3RBu0Tr Grateful for the support of @GlobalFund @USAID & Government of Germany – together we can #EndTB. @WHO Regional Office for Europe @stop TB partnership @Tereza Kasaeva

🔬 Exciting news in Stargardt disease research! Happy to share the latest breakthrough in our mission to combat Stargardt disease (STGD1). 🔬 Our recent scientific article presents the remarkable versatility of QR-1011, an antisense oligonucleotide (AON), initially designed to correct splicing in the most common severe variant associated with STGD1. Here, QR-1011 showed significant splice correction abilities for two additional severe STGD1 variants. Learn more about our study here: https://lnkd.in/ex3ut-VK This discovery offers the possibility for a broader therapeutic impact, potentially benefiting a larger group of STGD1 patients. Many thanks to all contributors involved in this achievement! #StargardtDisease #STGD1 #QR1011 #AON #ASO #rnatherapeutics #InheritedRetinalDisease

Research on new tools is fundamental if we are serious about TB elimination. The model we presented at the WHA 10 years ago when the End TB Strategy was endorsed foresaw the availability of shorter regimens, point-of-case tests for disease and infection, and a vaccine by 2025. Much more must be invested in research to reach targets and elimination.

Curious about the #SoHORegulation's impact on rare disease patients? Are you interested in the challenges to sustainable plasma supply in Europe and what can the #SoHORegulation do? Get insights from Lutz Bonacker, Senior Vice President and General Manager of Commercial Operations Europe at CSL Behring in this EURACTIV article. Check it out for a great read: 👉 https://lnkd.in/ekpfe5yb

The development of a new antibiotic is amazing achievement by Global Antibiotic R&D Partnership (GARDP), but comes with a warning about the risk of resistance as well as opening the potential for new diagnostics. #AMR #IP https://lnkd.in/gwfkkjUH

Do you know there are 7,000 rare diseases, and only 5% of them have a treatment? Let's join together to elevate awareness and advocate for improved treatment access for the millions of individuals around the world affected by rare diseases. Watch our special mini-documentary, which highlights impactful conversations from the World Orphan Drug Congress Europe 2023 - our attendees passionately discussed the transformative power of orphan drugs, sharing stories of hope, resilience, and breakthroughs in the face of adversity. #WODC #RareDiseaseDay #OrphanDrugs