Know Rare

09/30/2026

You’re invited to Rare in Motion! A free evening for people living with rare neuromuscular conditions, and the family and friends who support them.
• Learn exercises you can do at home
• Talk openly about the emotional side
• Hear what’s new in research
📅 Sun, Oct 18, 5–8 PM ET · 📍 Luma Studios, NYC, or free online. Register free: https://www.eventbrite.com/e/rare-in-motion-moving-forward-with-rare-neuromuscular-disease-tickets-2002235543409?aff=facebookorganic

Free hybrid event: adaptive exercise, emotional wellness and the latest research for rare neuromuscular disease. Join in NYC or online.

09/30/2026

Wondering whether your child's heart condition runs in the family? We're collecting questions from parents for a genetic counselor who specializes in heart conditions.

Is it inherited? Should siblings be screened? What does genetic testing actually tell you? No question is too basic.

Comment below with your question, and we'll share the answers soon.

Answers are general information and don't replace a consultation with your own care team.

09/29/2026

Just diagnosed with polycythemia vera, or supporting someone who is? These are the questions people search for most, answered in plain language. 💛

Swipe through, save it for later, and share it with someone who needs it.

You don't have to figure this out alone. Learn more at bit.ly/knowrare-pv.

08/28/2026

Living with Danon disease, or raising a son who is? We'd love to hear your story.

Know Rare is looking to connect with a man living with Danon disease, or a parent of a boy or young man with Danon, who'd be open to sharing what your journey has been like,, in your own words, about what life with Danon looks like day to day. The good moments, the hard ones, and everything you've learned along the way. Our hope is that other families navigating this feel a little less alone.

We're looking for someone who is US-based and English-speaking, and your time would be compensated.

If this sounds like you, we'd be so glad to hear from you. Please send us a DM if you're interested in participating.

08/17/2026

Thank you so much to for sharing what she wishes people knew about her life with ITP.

In her own words "I've been living with Chronic ITP for 7 years.
I want people to know that I'm more than a platelet count. That number can't show my exhaustion, the bruises covering my body, the extra heavy periods. It doesn't show how alone I felt as a teenager watching everyone else's world open up while mine seemed to be closing.

Life with a rare illness can be lonely, but having open conversations like this helps us to create an ITP community. If you're living with ITP, you're not the only one. 💜
I made video this with , who help connect people with real studies and researchers."

Visit https://bit.ly/itp-rhona to find out more!

08/07/2026

For parents of children with rare diseases, so much of daily life becomes about the roles: speech therapist, OT, PT, care coordinator, appointment scheduler, insurance advocate. But somewhere underneath all those hats is the most important one of all: just being mom. 💙

In our latest story, V.M. shares what it's really been like raising her son Jackson, diagnosed with a genetic duplication for 4p16.3p11. She opens up about the postpartum instincts that told her something was different, the long road to a diagnosis that sat in the uncertain middle ground, and the ongoing work of holding onto her identity as his mother through it all.

She describes grief as a balloon; inflating in the hard moments, deflating as you accept and move through each phase. She reminds us that grief isn't weakness. It's love.

To every parent learning to wear all the hats while still showing up as just mom or dad, whatever that looks like on any given day: we see you.

Read Jackson and V.M.'s full story: https://www.knowrare.com/blog-v2/learning-to-mother-a-child-with-a-rare-disease

07/15/2026

We celebrate another milestone in the clinical path to a potential treatment of Danon disease, a rare X-linked disease. Each stage of success brings the potential for treatment closer. To find out more about research studies in Danon disease, go to knowrare.com/danon.

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07/09/2026

If you live with ITP, you know the anxiety about the number, the next test, the what-ifs at 2am.

We were moved by writer Chris Anselmo's () honest piece on the Know Rare blog, "Anxiety: The Unwelcome Visitor," where he names his anxiety "The Vise", that slow, crushing pressure on the chest. Giving the fear a name doesn't make it silly. It makes it yours to manage.

So we pulled together a few tips for the specific worries that come with ITP: Name it. Ask your care team the hard questions. Build a calm kit. Breathe, and return to what matters.

And know that action is one of the strongest answers to fear. That's also why we talk about clinical studies; not as a last resort, but as a way to have more eyes on your counts, more frequent monitoring, and a hand in the future of ITP treatment.

Read Chris's full piece + explore research options: https://www.knowrare.com/es-US/blog-v2/anxiety-the-unwelcome-visitor-chris-anselmo

06/30/2026

Observational or interventional studies — what's the difference?

If you or someone you love is living with Danon Disease, you may have come across both kinds of clinical study. They sound similar, but they play very different roles in research.

Observational studies follow daily life and track the condition, without changing your current care. Interventional studies test a new intervention to see how it affects the way the condition is managed.

Both matter. The information gathered through observation today helps researchers design the therapy trials of tomorrow — and every story shared moves the whole community forward.

Curious whether a Danon Disease study might be right for you? We're here to help you understand your options, at your pace. Reach out anytime at https://knowrare.com/danon.

06/30/2026

“For a long time when I was first diagnosed, I wanted to ignore it. I pretty quickly learned that’s a good way to lose a finger.”

For Whitney , learning to live with scleroderma meant learning not to “tough it out.”

From protecting her hands and feet from the cold to preparing for air conditioning, moisturizing regularly, and addressing ulcers early, being proactive has become an important part of managing daily life.

Swipe through for Whitney’s personal tips for caring for her hands and navigating life with scleroderma.

Living with scleroderma and interested in research opportunities? Know Rare can help connect you with current studies and research.

Visit knowrare.com/scleroderma

Whitney is sharing her personal experience. This content is not medical advice. Always speak with your healthcare provider about your care.