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      Rare diseases

      Rare diseases

      Innovation
      A female scientist conducting research in a laboratory, focusing on orphan drugs for rare diseases

      What is an orphan drug?

      The need for these rare disease therapies is strong, and access to them can be lifesaving. Here’s how Johnson & Johnson is innovating to help give patients options.
      Caring & giving
      Sam Seavey piloting an aircraft from the cockpit

      3 health advocates; 1 Johnson & Johnson program that offers empowerment and connection

      In the quest to solve the toughest health challenges, innovating therapies and finding treatments are essential—but so is supporting the patients living with these diseases. That’s exactly why Johnson & Johnson launched HealtheVoices a decade ago.
      Health & wellness
      A newborn baby's feet with a hospital bracelet around its ankle

      What it’s like when your baby has a rare blood disorder

      A condition called fetal and neonatal alloimmune thrombocytopenia (FNAIT) can be deadly in newborns. That’s why Johnson & Johnson is passionate about developing a treatment for the disorder.
      Health & wellness
      O'Connor getting an infusion of medication to treat myasthenia gravis

      5 things we now know about myasthenia gravis

      For National Autoimmune Disease Awareness Month, discover more about the disease, learn how one woman lives with this rare autoantibody condition and why Johnson & Johnson is working to find new treatments for patients like her.
      Health & wellness
      An illustration of a crowd of people, all in black and white, with one person in the center wearing a red top and blue pants.

      What is a rare disease?

      It’s estimated that over 300 million people around the globe are living with a rare disease. For Rare Disease Day, here’s what to know.
      Personal stories
      A headshot of Hetal Patel, Medical Director, Global Medical Affairs, Immunodermatology at the Janssen Pharmaceutical Companies of Johnson & Johnson

      “My job is to educate about the need for new treatments for rare diseases”

      Hetal Patel, an immunodermatology medical director at Johnson & Johnson, forged her own career path—and now she’s living into her passion for advocacy, education and innovation.
      Innovation
      A view of a doctor from behind, as they look at brain scans

      The quest to make neurodegenerative disease a thing of the past

      Imagine reversing the course of conditions like Alzheimer’s or multiple sclerosis, in which cells of the central nervous system stop working or die. Researchers at Johnson & Johnson are hot on the trail of innovative solutions that may do just that, preventing often-debilitating symptoms along the way.
      Health & wellness
      Doctor holding hologram of human body

      4 things we now know about the rare disease AL amyloidosis

      Imagine having a disease that many doctors have never seen before. That’s what Marty Verel experienced when he was told he had AL amyloidosis—and it’s what hematologist Brendan Weiss, M.D., is determined to help change. For Rare Disease Day, Dr. Weiss shares what he’s learned studying the rare disease.
      Personal stories
      Doing Good for Rare Diseases Carl Guenther headshot

      “I have a very rare illness": The unique way patients and scientists are fighting Castleman disease

      Meet two men who have the rare immune system disorder: a doctor who created a groundbreaking registry for it—and a patient who signed up for it.