Charles Christensen+FollowThe $6 Million Bake Sale: One Mom’s FightImagine knowing there’s a cure for your kid’s rare disease—but it’s locked behind a $6 million paywall. That’s Catherine Illingworth’s reality. She’s hustling on TikTok, organizing bake sales, and rallying her network, all to fund a treatment that’s just out of reach. Turns out, thousands of families are in the same boat, forced to become full-time advocates and fundraisers just to give their kids a shot at life. The system? Totally broken. #Health #BodyHealth #RareDisease20Share
Henry Chan+FollowNKY family rallies for rare disease awarenessOnly 50 kids worldwide share Van Foster’s rare KARS mutation, and one of them lives right here in Northern Kentucky. Van’s parents are turning heartbreak into hope, organizing a volleyball fundraiser to support research and connect with other families facing the unknown. Every milestone Van reaches is a huge victory, given the challenges he faces daily. Their story is a powerful reminder of how community support and advocacy can drive change for rare diseases. Would you join a local event like this to help families in need? #News #RareDisease #CommunitySupport00Share
kerryrivera+FollowChapel Hill family’s fight sparks hope for rare diseaseA Chapel Hill family turned heartbreak into action after both their twin daughters were diagnosed with Juvenile Batten disease, a rare and devastating genetic disorder. Instead of giving up, the Kahns launched the ForeBatten Foundation, raising over $8 million to fund research and support others facing this diagnosis. Their efforts led to a groundbreaking, first-of-its-kind drug trial at UNC—tailored specifically for their girls. While there’s still no cure, the progress has brought moments of joy and hope back into their lives. How do you think communities can best support families battling rare diseases? #Safety #ChapelHill #RareDisease20Share
Jacqueline Howard DDS+FollowShe Flew 4,000 Miles to Save Her LifeImagine being told you’re slowly starving to death—and the only hope is a surgery 4,000 miles away. That’s what happened to Kristina, who was diagnosed with a super rare illness (MALS) that barely anyone gets. US doctors couldn’t help, so she and her husband flew to Germany, where surgeons found even more rare conditions and saved her life just in time. Two years later, she’s finally healthy, eating again, and sharing her story to help others fighting rare diseases. Talk about a comeback! #Health #BodyHealth #RareDisease50Share
Joseph Bradley+FollowThis Teen Can’t Eat ANY Food?!Imagine being allergic to literally every food out there. That’s Finley’s reality—his body treats all food like a virus and even bleeds internally if he tries to eat. Doctors are totally stumped and haven’t found anyone else like him. He gets all his nutrients through tubes straight to his heart and stomach, and the condition is so rare, it doesn’t even have a name (he calls it 'Finley-itis'). Wild, right? #RareDisease #MedicalMystery #Unbelievable #Health150Share
Aaron Rodriguez+FollowGame-Changer for Rare Swelling DisorderBig news for anyone dealing with hereditary angioedema (HAE): the FDA just approved Andembry, a once-a-month shot that actually prevents those scary swelling attacks before they start. It’s the first drug to target the real culprit (factor 12) and people in trials had way fewer attacks—like less than one a month! Plus, it’s got fewer side effects than older treatments. If you or someone you know struggles with HAE, this could be a total life upgrade. #HAE #RareDisease #FDAApproved #Health #BodyHealth30Share
rachel60+FollowBaby’s Life Saved by Custom Gene TherapyA baby born with a super rare, deadly disorder just became the first person ever to get a one-of-a-kind, personalized gene therapy—and it’s working! KJ Muldoon was facing a future of strict diets, risky transplants, and scary odds, but thanks to a CRISPR-based treatment made just for him, he’s now thriving and hitting milestones. Even when he got sick (which usually means disaster for kids like him), he bounced back like a champ. This could totally change the game for rare diseases! #GeneTherapy #MedicalBreakthrough #CRISPR #RareDisease #ScienceNews #Health190Share
Mr. Roy Ewing II+FollowGene Editing Just Saved This Baby’s LifeA one-in-a-million baby, KJ Muldoon, was born with a rare genetic disorder that could’ve been deadly. But thanks to a custom CRISPR gene editing treatment made just for him, he’s now thriving! Instead of a risky liver transplant, doctors used a new “base editing” technique to fix his DNA. He’s hitting milestones his family never thought possible. It’s early days, but this could totally change the game for rare diseases! #GeneEditing #CRISPR #MedicalBreakthrough #RareDisease #ScienceNews #Health891Share
Aaron Rodriguez+FollowRare Cancer Drug Gets Fast Track NodBig news for anyone following cancer breakthroughs: the FDA just gave Fast Track status to givinostat, a potential game-changer for people with polycythemia vera (PV), a rare blood cancer with few treatment options. This means the drug could reach patients faster if trials go well. Givinostat targets the root cause of PV and could help control symptoms and lower the risk of serious complications. Fingers crossed for the Phase III trial! #CancerResearch #FDA #RareDisease #MedicalBreakthrough #PolycythemiaVera #Health2325Share
Michael Flores+FollowNew Molecule Could Change Rare Disease GameScientists just found a single molecule that might actually reverse the effects of rare, devastating genetic diseases caused by POLG mutations. This is huge—these conditions are tough to treat and can show up at any age. The new compound, PZL-A, basically helps the cell’s powerhouses (mitochondria) fix themselves, even when their DNA is messed up. Early lab results are super promising, and clinical trials are already underway. Could this be the breakthrough so many have been waiting for? #ScienceNews #MedicalBreakthrough #Genetics #RareDisease #HealthInnovation #Science90Share
