Resources & Articles

My experience with Leigh syndrome has opened my eyes to how many people are impacted by rare disease — and what we can do about it.

People I meet ask me what I do and I say, “I write children’s books for kids with sickle cell disease.”

2021 was another exciting year at PicnicHealth. We’re looking back and celebrating all that we accomplished with our patient volunteers and life science partners. Take a look with us. And thank you for coming along on our journey.

The same goes for NPC research — we need community participation to move it forward.

This year, the theme focuses on Black Health and Wellness — an especially timely focus.

I’ve realized how much families like mine can help families currently dealing with the everyday challenges of NPC.

As the founder of Myositis Support and Understanding Association (MSU), I’ve seen and experienced how far-ranging the impact of living with a rare disease like dermatomyositis can be.

Because of what I’ve been through as a rare patient, I’m driven to help people facing similar challenges.

Having dermatomyositis is life-changing, and you never know what your new normal is going to be.

A conversation with three AllStripes Ambassadors for the ALD research program.

After my son Lincoln was diagnosed, I started connecting with others all over the world about their PROS journeys.

Matthew and his older brother, Michael, enjoy goofing around together — just ask them what happened with a name tag at the National Institutes of Health!

As the founder of Sickle Cell Reproductive Health Education Directive, I’m fighting to end disparities and offer resources I wish I'd been able to access earlier on my infertility journey.

It’s an exciting time for Alzheimer’s disease research. The FDA approved the first new drug for Alzheimer’s in nearly 20 years in June 2021, and other potential treatments, targeting amyloid plaques in the brain, are making their way through clinical trials.1, 2 It’s a glimmer of hope for Alzheimer’s patients, who number over 6 million in the U.S. alone.

Bethany, the co-founder of the Allo Hope Foundation, describes how her experience with alloimmunization and HDFN changed her life forever.

Life with Roya means our family has to just let go a little bit and be in the present.

Talking to other parents in the IRF2BPL community might not immediately change our situation, but it does validate our family’s experience with this ultra rare condition.

PicnicHealth is excited to announce our Holiday 2021 Donation Campaign. PicnicHealth is donating to patient advocacy groups everytime a patient joins a PicnicHealth research study until January 2, 2022.

Let’s face it: There’s a lot to manage when you embark on a caregiving journey for someone with Alzheimer’s disease. Whether you’re just starting out or you’re well along the path, there are symptoms to monitor, appointments to navigate, medications to manage, and a whole lot more. It’s easy to become overwhelmed—but a few strategic moves can go a long way toward alleviating stress and lightening your caregiver load. The following five tips can be your guide to supporting your loved one—and yourself—through the twists and turns of Alzheimer’s disease.

Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) are a group of rare conditions that impair the body’s metabolism from breaking down certain fats from food into energy. Although there are an estimated 2,000-3,5000 people living with LC-FAOD in the U.S., researchers have limited data on how LC-FAOD progresses overtime and how it is managed in the real world.

Haley loves singing and horses, but watching little pieces of her fall away has been hard. It’s why we founded the Haley’s Heroes Foundation.

Listen to Sylvia and Mubina explain how they bonded for life over caring for their mothers, who both had the rare disease progressive supranuclear palsy.

These days, I'm managing a liver transplant rather than CN1.

We’re excited to announce the formation of our Scientific Advisory Board (SAB) and welcome inaugural members, including Robert Kaplan, PhD, Jeffrey Brown, PhD, and Milena Gianfrancesco, PhD, MPH.

PicnicHealth is excited to partner with Komodo Health to enrich its patient-centered real-world data Research Platform. This collaboration will enable PicnicHealth to unlock rich new insights about complex diseases and treatment outcomes.

The esteemed pediatric neurologist spoke to AllStripes about Niemann-Pick disease type C research, the importance of medical records — and hope.

Four members of the rare disease community describe their experiences with coordinated health care.

“There are times when it’s not fun, but there are benefits you’ll see once you accept the diagnosis and begin to meet others.”

A roundup of thoughtful and energetic takeaways, courtesy of our AllStripes Ambassadors.

Lara is the mother of Eden, a bubbly 13-year-old with a rare condition. After years of symptoms, Eden was diagnosed with Kleefstra syndrome at 8 years old.

Community builder Ade Adeyokunnu refuses to let sickle cell keep him from living.

Some basic information for anyone with 504s or IEPs on their mind this back-to-school season.

This week, we’re exploring just a few of grief’s many forms.

Blaine has fully embraced the community that helped him through the early years of his diagnosis with IBM, a rare muscle disease.

Only 5 percent of rare diseases have approved treatments. How could drug repurposing help?

Barry is a gregarious, outgoing jokester. He’s also leading the charge for adult GM2 research.

This week, Finding Dory and disability representation, how the time tax relates to rare disease and profound insight on giving and receiving support.

After Alexis began experiencing eye pain, a doctor predicted she had a long diagnostic journey in front of her. He was right.

Content about disabilities, curated with the rare disease community in mind.

To me, they mean self-confidence, loving my disabled body fully and unapologetically taking up space.

After Dinah’s daughter was diagnosed with CCM3, a rare condition that causes brain lesions, she went looking for clues about what might lie ahead.

Julie’s daughter Amber, who has Kleefstra syndrome, went through a period of regression that stumped her doctors.

Karen would rather people take a straightforward interest in Doug than stare at him.

How Julianne, a young adult with Gorham-Stout disease, is pushing past her own discomfort to help others.

Joe is determined to help his family, one meal at a time.

How making a friend in the rare-disease community led to an accurate diagnosis of PROS.

Andrea was rejected for professional dance jobs because PROS caused growths to develop on her face, but she refused to let society’s standard of beauty stop her from living out her passion.

Scott was diagnosed with IBM four years ago, but works hard to keep doing the things he enjoyed before.

When Shaye first learned she and her baby were at risk for complications, she felt confused and devastated. Then she found others who'd been in her shoes.

When Elizabeth went to a doctor to treat her sore throat, she discovered that it was actually a symptom of a rare disease she’d unknowingly been living with her whole life.