10 results for "rare disease"
🧘 Health & WellnessOHSU Scientists Find New Target for Brain Autoimmune Disease
Scientists at Oregon Health & Science University have discovered exactly where harmful antibodies attack the brain in a rare but devastating disease. The breakthrough could lead to new treatments and an early detection blood test for anti-NMDAR encephalitis, which primarily strikes people in their 20s and 30s.
🧘 Health & WellnessSarcoma Survivor Helps 9,000 Patients Fight Rare Cancer
After surviving a rare and misunderstood cancer twice, one man built a support network that has reached 9,000 sarcoma patients across India. His mission: ensure no one fights this lonely battle alone.
🧘 Health & WellnessFDA Grants Breakthrough Orphan Drug Status for Rare Blood Cancer Treatment
The FDA has awarded Orphan Drug Designation to CK0804, a promising new treatment for myelofibrosis, offering renewed hope to patients battling this rare and challenging blood cancer. This special designation will accelerate development and bring the therapy closer to patients who desperately need new treatment options.
🎬 VideosAurora Brings Hope to Millions Living with Rare Diseases Through AI Innovation
Healthcare innovator Aurora is harnessing the power of artificial intelligence to develop life-changing therapies for the millions of patients worldwide who suffer from rare diseases. Backed by leading venture capital firm Menlo Ventures, this groundbreaking approach promises to bring treatment options to those who have long been overlooked by traditional pharmaceutical development.
🧘 Health & WellnessNew Cancer Treatment Gives Pancreatic Patients Years of Hope Beyond Expectations
A groundbreaking therapy is helping patients with rare pancreatic and bile duct cancers live years longer than expected, with 65% achieving long-term disease control. Some patients have thrived on the treatment for over three years, offering new hope for hard-to-treat cancers.
💛 Acts of KindnessBluffdale Family Turns Rare Disease Battle Into Hope for Others
A Bluffdale family with two children facing a rare creatine deficiency has transformed their personal struggle into a mission of hope. The Wallis family is now funding research and helping others by selling high-quality creatine, turning each purchase into progress toward a cure.
🧘 Health & WellnessBarcelona Scientists Develop Breakthrough Method to Design Precision Drugs for Fatal Disease
An international research team led by Barcelona scientists has unveiled a revolutionary approach to fighting transthyretin amyloidosis, opening doors to personalized treatments for patients with this fatal condition. Their groundbreaking method captures protein behavior like a movie instead of a snapshot, transforming how doctors can target rare genetic diseases.
💛 Acts of KindnessBeaumont Family Raises $1M for Rare Disease Research Through WhiskeyFest
After their 2-year-old son Hudson was diagnosed with Duchenne muscular dystrophy, Mike and Katherine Williams transformed their love of whiskey into a powerful force for good. Their annual Southeast Texas WhiskeyFest has raised over $1 million in just four years to fund cutting-edge research for rare diseases that often get overlooked.
🚀 InnovationNobel Winner Jennifer Doudna Launches Startup to Save Kids with Rare Diseases
A revolutionary new company co-founded by gene-editing pioneer Jennifer Doudna is bringing hope to thousands of families facing rare genetic diseases. After a groundbreaking treatment saved baby KJ's life in just six months, Aurora Therapeutics is scaling this life-saving technology to help many more children who previously had no options.
🚀 InnovationBerkeley Scientist Launches CRISPR Startup to Bring Personalized Gene Therapy to Rare Disease Patients
A key scientist from the groundbreaking Baby KJ gene editing success story is turning hope into action by launching a new company dedicated to making personalized CRISPR treatments accessible to patients with ultra-rare genetic conditions. The innovative startup aims to scale life-saving gene therapies that were once thought impossible to deliver broadly.