Know Rare

🎙️NOW STREAMING: Rare Insights A new podcast series focusing on the future of potential rare disease treatments. 🧬 This series promises to delve deep into the cutting-edge research and innovative therapies that are shaping the landscape of rare diseases. Industry and organizational leaders share the latest advancements, providing expert perspectives and valuable knowledge. These thought leaders will discuss breakthroughs and the impact these innovations may have on patients’ lives and the healthcare industry as a whole. Hosted by Taren Grom, co-founder and former media executive of PharmaVoice, this podcast brings unmatched expertise and insight to the conversation. Grom’s experience and passion for the subject will guide you through each episode, ensuring a rich and informative experience. ✨LISTEN NOW✨ wherever you stream your podcasts! For more information visit: https://lnkd.in/ggxKiXws - #medicalresearch #raredisease #rarediseaseawareness #clinicaltrials #clinicaltrial #healthcareindustry #clinicalresearch #KnowRare #RareInsightsPodcast

Know Rare can help you connect to specialists studying potential new treatments and options for those diagnosed with Multifocal Motor Neuropathy (MMN)    🔗 Learn more by visiting https://lnkd.in/guJFz47H    -    #MultifocalMotorNeuropathy #MMN #muscleweakness #clinicalstrials #clinicaltrial #raredisease #rarediseases #rarediseaseawareness #rarediseaseadvocacy #KnowRare

🎙️LISTEN NOW: Transformative Cell and Exosome-Based Therapies with Capricor Therapeutics https://lnkd.in/graea4ew Discover how cutting-edge cell and exosome-based treatments are paving the way for new hope in Duchenne muscular dystrophy and beyond with Dr. Linda Marbán, CEO of Capricor Therapeutics, Inc. Join us as Dr. Marbán discusses: • CAP-1002’s journey through Phase 3 trials. • The game-changing role of exosome technology. • What the future holds for rare disease therapeutics. • Key shifts in the rare disease space over the years. • Her unwavering dedication to the rare disease community. Listen to episode 3 “Transformative Cell and Exosome-Based Therapies with Capricor Therapeutics” NOW! 🔎 Just search “Rare Insights.” - #RareInsightsPodcast #medicalpodcast #raredisease #rarediseaseawareness #rarediseaseresearch #medicalresearch #medicalinnovation #DuchenneMuscularDystrophy #CellTherapy #ExosomeTechnology

Share Your Rare: Kathleen's Story 💙    After more than a year without having a diagnosis, Kathleen’s family was relieved when they finally received one for their child Noah, but they also felt like their world was crashing down. Noah was diagnosed with PPA2, a rare genetic condition that can cause sudden cardiac arrest in infants.     One of the biggest challenges that Noah overcame was a 25-day stay at a pediatric intensive care unit that involved three cardiac arrests and a week-long intubation. Fortunately, Noah was able to re-learn how to eat, talk, sit, and walk, with minimal outside help.    Noah's doctors always say that he isn't their typical mitochondrial/metabolic disease case. He seems to be growing normally, looking and acting like a typical 3-year-old. However, he's had four cardiac arrests, more hospital visits than the family can count, has a pacemaker, and is on an extremely restricted diet.    Kathleen is appreciative of their amazing team of doctors, but, above all, she is so thankful for all the families that she has connected with who are also living with PPA2. She is forever grateful for their incredible support.    Kathleen’s family hopes to continue building a caring community, spreading awareness, and sparking interest in research and treatment options. They also would like to help people understand the severity of this rare disease. What may seem like a simple cold to one person, may lead to death for Noah. Therefore, it is crucial to be mindful of people with suppressed immune systems and take the right precautions.    -    #raredisease #rarediseaseawareness #rarediseases #rarediseaseadvocacy #KnowRare 

🎧 Coming Soon: Transformative Cell and Exosome-Based Therapies with Capricor Therapeutics Discover how cutting-edge cell and exosome-based treatments are paving the way for new hope in Duchenne muscular dystrophy and beyond with Dr. Linda Marbán, CEO of Capricor Therapeutics, Inc. Join us as Dr. Marbán discusses: • CAP-1002’s journey through Phase 3 trials. • The game-changing role of exosome technology. • What the future holds for rare disease therapeutics. • Key shifts in the rare disease space over the years. • Her unwavering dedication to the rare disease community. Tune in on August 26th to listen to episode 3 "Transformative Cell and Exosome-Based Therapies with Capricor Therapeutics". Meanwhile, catch up on all our episodes on Apple Podcasts, Spotify, or your go-to podcast platform. 🎙️ Just search "Rare Insights." - #RareInsightsPodcast #medicalpodcast #raredisease #rarediseaseawareness #rarediseaseresearch #medicalresearch #medicalinnovation #DuchenneMuscularDystrophy #CellTherapy #ExosomeTechnology

Help researchers learn more about your life with Congenital Myasthenic Syndromes (CMS) 💙    No one knows more about CMS than people who live with it. Share your experiences with the leading researchers in CMS to help them develop potential new ways to treat it.    🔗 Learn more by sending us a DM or visiting https://lnkd.in/gptTN739     -    #CongenitalMyasthenicSyndromes #CMS #eyedisease #raredisease #rarediseases #rarediseaseawareness #medicalresearch #clinicaltrials #clinicaltrial #KnowRare

Get ready to explore the future of rare disease treatment!     In the upcoming episode 3 of "Rare Insights," airing on August 26th, host Taren Grom chats with Dr. Linda Marbán, CEO of Capricor Therapeutics, Inc.. Dive into the groundbreaking world of cell and exosome-based therapies that are on track to revolutionize treatments for Duchenne muscular dystrophy and other rare diseases. 🌟💊    Dr. Marbán sheds light on:   • The progress of CAP-1002 in Phase 3 trials for Duchenne muscular dystrophy.   • The unique power of exosome technology in rare disease therapeutics.   • The future of cell-based treatments and what’s on the horizon.   • Major shifts in the rare disease sector since 2005.   • Her deep commitment and passion for the rare disease community.    Tune in on August 26th to listen to episode 3 Transformative Cell and Exosome-Based Therapies with Capricor Therapeutics.    Meanwhile, catch up on all our episodes on Apple Podcasts, Spotify, or your go-to podcast platform.     🎙️ Just search "Rare Insights."    #RareInsights #Podcast #DuchenneMuscularDystrophy #CellTherapy #ExosomeTechnology #RareDiseases #MedicalInnovation

An introduction by Laura Will...    "When I stumbled upon Carolyn’s Facebook page about her journey caring for her son Alvin, it felt like the distance between Kenya and my home office on the East Coast of America collapsed.    Her struggles and worries, the fumbling and uncertainty, the exhaustion and strength were so closely mirrored in my rare-mom heart, I felt I could reach out just beyond the screen in front of me, and she would be there. Her story, and the story of her son Alvin, are still evolving. The tireless work to give her son a life of dignity and health meets both new and ongoing challenges.    She has recently decided to share her story more widely, and we here at Know Rare are lucky enough to highlight this rare mom as she starts to write her own narrative."    🔗cRead 'A Day at a Time: Our Journey with Rare Disease and Relentless Seizures' on The Know Rare Blog! https://lnkd.in/evjwxJka    -    #specialneedsparent #spooniesupport #epilepsystrong #spooniestrong #neurologicaldisorder #spoonielife #invisibledisease #invisibleillnessawareness #raredisease #rarediseaseawareness #KnowRare

The Propionic Acidemia Foundation is a 501(c)3 non-profit organization dedicated to finding improved treatments and a cure for Propionic Acidemia by funding research and providing information and support to families and medical professionals.    The PA Foundation’s vision is to create a future where Propionic Acidemia can be prevented and any affected individual can be cured and live a productive life.    🔗 Read more on The Propionic Acidemia Foundation on The Know Rare Blog! https://lnkd.in/g5XrMJzA    -    #PropionicAcidemiaFoundation #PropionicAcidemia #acidemia #raredisease #rarediseaseawareness #rarediseaseadvocacy #medicalresearch #clinicalresearch #KnowRare

"On April 28, 2008, a doctor I didn't know walked into recovery and said, “You have cancer. It's terminal.”    Upon hearing the news, my sister, head of Radiology at Fletcher-Allen Hospital, Burlington, Vermont, told me to speak with Doctor Thomas Roland, Head of Radiology. Half an hour later, he called. We spoke for forty-five minutes. After that call, I drove through three states to arrive at his office at six in the morning. My sense of urgency was met with a promising development: over ten hours, we developed a program that included a trial drug, Mitomycin 5fu.     I told him I had nothing to lose, and maybe someone else would have a chance. Cancer runs rampant in my family: I've lost all the major players except my maternal grandfather. I have ten siblings, five of whom have had cancer. Thirteen different types.    I had been carrying that cancer inside me for four years, dealing with extreme fatigue and unbelievable constipation, though nothing else. Within days I was having side effects I didn't know existed, including an immediate allergic reaction to the Mitomycin 5fu.     My daily appointments were for 5:30 in the morning. That time is when small children typically were in the hospital for treatment. It was agonizing—I watched them die and could do nothing. I am no hero, but I am a mother, sister, and friend. As I went through the treatments and saw what happened to those young lives, my quest changed. My motto became “For The Children.”     “I know I was only one among many, but I felt elated. All I had gone through was suddenly validated.”"    🔗 Read the full story 'My Unexpected and Powerful Clinical Trial Journey' on The Know Rare Blog: https://lnkd.in/ghjH5-2y    -    #rarecancer #raredisease #rarediseaseawareness #spoonies #spooniestrong #curecancer #cancerfighter #cancercommunity #cancerwarriors #KnowRare