Sanofi’s Post

Collaborating as a team can significantly impact #rarediseases, whether through #advocacy and #awareness, patient-centred care, or #research and innovation. When it comes to rare diseases, we are stronger together, and it's great to have you on the team! Throughout February we ran a ‘For The Love Of CACNA1C’ campaign on our social media channels to continue to raise awareness of our rare disease. We worked together as a community to create personal messages of hope and support to signpost #CACNA1C individuals, families and carers who may not have found us yet. With your support, we can spread the word far and wide. The growth of our community increases knowledge and understanding of our disease-causing gene, we are dependent on it. Did the ‘For The Love Of CACNA1C’ campaign work? It totally did. I've created a graph below to illustrate the impact you have made from supporting our social media channels. The pink colour represents the months of this year to date. Look at the numbers for March following the February campaign! Throughout April, we launched another campaign, this one involved close #collaboration and planning with our families in Brazil to ensure we accurately represented the stories and messages they wanted to share. All our posts during April were in Brazilian Portuguese as well as English. Look at the numbers for May and it’s only the 22nd! We are delighted to share that the growth in May includes a family in Brazil, meaning we are now in contact with, and able to support, 8 CACNA1C families in Brazil. As our community expands, real-world healthcare data can be converted to evidence through the CACNA1C Community Registry (which currently has 72 participants) to accelerate and support clinical and basic research. Teamwork does make the dream work. Thank you for all your support. You are making a difference. #RareDisease #LanguageAccess #PatientRegistry #RealWorldData #PatientEngagement #TimothySyndrome #LongQT8

There are 12.4M people worldwide who are affected by the most common type of polycystic kidney disease (PKD) called Autosomal Dominant (ADPKD). Autosomal Recessive is a rarer form that occurs in 1 in 25,000 children worldwide. Neither of these chronic kidney diseases can be cured. Otsuka’s perseverance in developing treatments for people living with kidney disease has led to breakthroughs that have helped change the landscape of treatment options and set the stage for other new developments in nephrology. In addition to drug development, we have spent years advocating on behalf of patients through programs and institutions focused on kidney disease. We will continue to serve our patients and clinicians each and every day, so they can achieve more than they thought was possible. Help spread awareness about PKD by learning more here: https://bit.ly/3QOhmAY https://bit.ly/4cSEHsA

Remember when they told us that one glass of red wine a day was good for you? New research connects moderate drinking (1 or fewer a day for women, 2 or fewer a day for men) to increased risks of cancer. Big yikes. https://lnkd.in/gYWXJUa9 So how can we all, with the knowledge we have, help re-set workplace culture that doesn't center on alcohol? Can we commit to providing an NA choice at every function? To supporting sober employees find and connect with each other to navigate the next in person company event (that's often filled with alcohol)? Sobrynth is here to support employees name and reach their sober goals, while also providing best practices to employers to ensure we're creating supportive, inclusive environments for our sober and sober curious colleagues. Let's chat. This work is going to take all of us, whether you're sober or not. We need sober allies to help further this mission. #recoveryfriendlyworkplaces #makeNAnormal #alcoholandcancer

What an incredibly powerful way to talk about a benefit, policy or value that you share as a company. I was really moved, inspired and emotional watching this film from Sanofi detailing their commitment to removing the stigma of cancer at work. Cancer & Work: Acting Together is their 360° approach to addressing the financial, emotional, social and professional needs of colleagues impacted by cancer or other critical illnesses. Definitely give it a watch and stay to the end. An inspiring message and a creative approach to learn from to get your audience thinking and engaging in an unexpected way. #employerbranding #employerbrand #wellbeing #benefits #values

Did you know that ANZCHOG currently sponsors 25 trials that are open or preparing to open? 😲 Our Clinical Trials Wheel is a great way to see what trials are currently open or preparing to open. The centre circle and different colours detail if the trial is open to newly diagnosed or relapsed patients, or in some circumstances where a trial is open to both newly diagnosed and relapsed patients. Moving outwards, the second circle shows the different disease types that a trial is relevant to and the outermost circle shows the name or acronym of each trial. More information on any of these trials can be found on our website, anzchog.org by simply searching the trial name or acronym. ANZCHOG is consistently working to open new local and international clinical trials for Australian and New Zealand children, adolescents and young adults, with an additional 15 + trials currently in the feasibility phase. These trials wouldn’t be possible without our generous supporters and our amazing clinical trials team. Thank you 💙💚 #ANZCHOG #WorldClassCancerCare #ClinicalTrials #PaediatricOncology

The first time I watched this video, it truly touched me. I couldn’t help but think of my loved ones, my friends, my mother... I felt I had to share it with you. When someone we care about is sick, how do we manage to stay focused at work? And what if we need help to deal with it? When Cancer or Critical Illness affects your life, the last thing you should be worrying about is work. This October, we’re celebrating Cancer & Work: Acting Together - our 360° global benefit, offering physical, mental, financial, and social support to all of us through the toughest of times. At Sanofi, we provide time off for recovery and safe spaces to connect and learn from each other’s experiences. Whether you’re a manager or colleague, whether you’ve had cancer or cared for someone with critical illness, you’re not alone. Cancer & Work affects us all. Learn more about our Cancer & Work program : http://spkl.io/6040fRSsa #CancerAwareness #Care

In one instant, your life is turned upside down. A small accident, a slight stutter, a mild discomfort proves to be something much bigger. At that point, there is no turning back. On 29th February, Rare Diseases Day helped us remember how #lifesciences and #biotech professionals have a key role to play helping cure rare diseases. A rare disease is defined as a condition that affects less than 200,000 people in the USA and fewer than 1 in 2,000 people in the EU. In the past, the development of orphan drugs was made difficult due to the small size of patient populations, which acted as a barrier for commercial development in the research and development of rare diseases. Yet, a rare diseases, especially when it affects children, has been proven to have a huge societal and economic toll. To address this disconnect, significant IP, regulatory and commercial incentives have been implemented to accelerate development of new treatments, first in the US (1983), in Japan (1993) and finally in the EU (2000). Treatments have been brought to market and more will come. (see this recap paper of key late-stage pipeline developments from IQVIA ➡ https://lnkd.in/daf3fudb) Beyond these incentives, the development of rare diseases have been pushed forward significantly by one strong tailwind: the unwavering commitment of patients community. Patients affected by rare diseases may be few but the families and communities affected are extremely large... and extremely vocal. In the US, we saw the strength of such communities around the approval of novel treatments for Duchenne Muscular Dystrophy by Sarepta Therapeutics. The fight continues. In France , the AFM-Téléthon has been unrelentingly advocating public authorities for years to support the development of novel treatments. It is today one of the most popular association in France. Today, with the support of Bpifrance and other key public and private stakeholders, it is building a very comprehensive #biotech ecosystem of public research institutes, investors, startup studios and biopharma players to further accelerate this effort. This is today. There is no turning back. ✊ #rarediseasesday #rarediseases #orphandiseases #pediatrics #businessdevelopment #patientadvocacy https://lnkd.in/dQ7kD43E

Clinical trial participation has been proven repeatedly to improve patient outcomes by providing access to new therapies and more frequent monitoring. The World Pancreatic Cancer Coalition recommends that every person be given a chance to enrol in a clinical trial. Numerous success stories underscore the potential benefits of clinical trials; however, Canadian participation remains alarmingly low. According to the University of Windsor, only about 7% of Canadians with cancer participate in clinical trials, and this figure drops by almost half in smaller provinces and rural areas (Sharma et al., 2022). In contrast, major centers like Princess Margaret Hospital in Toronto achieve around 21% engagement. This low engagement hinders Canada’s ability to advance cancer treatment and improve its stagnant survival rates.

The Los Angeles Times has written a powerful story on the need for California Institute for Regenerative Medicine (CIRM) to continue allocating at least 50% of funding for rare disease. My two cents: 1. Abla Creasey, Vice President at CIRM (and wonderful patient advocate) mentions that funding will exist for rare but may decrease and that applications for large diseases and rare ones will be treated equally. To me this is a major flaw. Taxpayer dollars should go to public health needs where the capital markets are not working. Large diseases are already massively funded by pharma and federal taxes. They don’t need our state tax dollars. 2. I have heard rumors that CIRM may take a “platform approach” to rare. I am worried what this means. We know that rare diseases get cured when patient advocacy groups or small biotechs are given funding. There are plenty of cell and gene therapy platforms available - I truly hope funding will still go to the organizations taking the risk and also are able to efficiently develop drugs.

For families in the rare disease community, the path to a diagnosis can be a long and challenging journey, often taking years to reach an accurate conclusion. But we're determined to change that. In August 2022, we launched our Early Detection and Diagnostic Committee with a clear mission: to cut down the time it takes to diagnose TANGO2 deficiency disorder (TDD) and ensure that families get the care and support they need, sooner. This dedicated team has been tirelessly advocating for faster, more accurate diagnoses and making remarkable strides along the way. Here’s what we’ve accomplished so far: ✅ Successfully advocated for the inclusion of TDD on gene panels, speeding up the diagnostic process. ✅ Ensured accurate, clear language about TDD on various medical websites, improving awareness and understanding. ✅ Co-authored and branded a critical TDD resource for the Hispanic community in Mexico, broadening our impact. ✅ Expanded our committee from 4 to 9 passionate individuals, strengthening our efforts and outreach. ✅ Participated in Global Genes’ Rare Disease Mentoring program, connecting with other leaders in rare disease advocacy. Every milestone brings us closer to our goal: saving lives, improving quality of life, and making the diagnostic journey shorter and less daunting for every family affected by TDD. Together, we’re making a difference, one breakthrough at a time. 💜 #TANGO2ResearchFoundation #EarlyDetection #RareDiseaseAwareness #TANGO2Community #HopeForRareDiseases