I just finished watching a screening, and I don’t think I’ve ever experienced anything quite like it. I cried—not just once, but throughout—and not only from sadness, but from a deep, overwhelming sense of connection.
As a deep empath, I often feel the world intensely. But this film was different. It held both the weight of rare disease and the light of hope in the same breath. They gave voice to the unspeakable, and dignity to the invisible. It was beautifully human, heartbreakingly honest, and profoundly inspiring.
What struck me most was how they wove together science and soul—showing not just what rare disease is, but what it means to the families, the researchers, and the people in between. You made it personal. You made it real. And through that, you made it impossible to look away.
Thank you for creating this. For honoring these stories. For sparking the kind of conversation that the rare disease community has needed for far too long.
💔 Rare disease is not rare.
It’s not a niche problem—it’s a silent global crisis.
🧬 300 million affected globally | 1 in 10 in the U.S.
🧒 50% are children
⏳ Average 5–7 years to diagnosis
💊 95% have no treatment
💸 Families often fund research themselves
RARE isn’t about tragedy—it’s about power.
It’s about families driving the search for cures, funding research, building foundations—because no one else will.
This film is a window into the future of medicine, and a mirror of our values.
If we believe every child deserves a chance, rare disease can no longer live in the shadows.
Jennifer Gehring
Read more at: linkedin.com
Last night I caught a screening of the greatest superhero movie I have ever seen.
I sat in the audience watching my favorite rockstars on the big screen, shedding tears for their struggles and on the edge of my seat as they stepped forward into battle scenes.
The superheroes here were rare disease patients and parents and their pioneering research allies, racing the clock for their kids. Their weapons included for battle were gene therapies, ASOs and repurposing molecules along with smarter business models to fight in indications too small for most companies to notice.
Assembling this ensemble was Lainey Moseley (rare disease mom and documentary filmmaker) -- the Nick Fury of the evening. And the superheros I was cheering on included Casey McPherson (AlphaRose Therapeutics | To Cure A Rose Foundation), Pat Furlong (Parent Project Muscular Dystrophy), David Fajgenbaum, (Every Cure), Ethan Perlstein (Perlara PBC), Sunitha Malepati (Buffalo Initiative | CACNA1A Foundation) and Cat Lutz (Jackson Laboratories). I was so excited that I did sneak some photos during the movie, but I did seek forgiveness from Lainey!
Grateful to the Montclair Film Festival for featuring the screening with support from Sarepta Therapeutics and outstanding panel moderation with Wendy Erler.
This is a powerful film to bring for screenings to your company, your community, or your next conference. "Rare" has captured and documented a transformational movement on the cusp of creating sustaining breakthroughs for the 30 million Americans and 300 million around the globe facing rare disease diagnoses without meaningful treatment options.
Craig lispet
Read more at: linkedin.com
Rewatching Lainey Moseley’s Independent Documentary – RARE: A Rare Disease Revolution
I had an opportunity to rewatch this incredible film over the weekend, and share the viewing experience with my wife. This second time watching, with Dr. Kingsmore’s quote in my head, it carried such a different perspective.
The families in the documentary know their enemy, thanks to sequencing and screening. Knowing your enemy is the first building block to galvanize support, build a community, and direct action towards a cure. The RARE documentary doesn’t call these families’ journeys “therapeutic odysseys”, but it’s precisely at the core of the #TooRareToCare thesis that rips at your heartstrings. Showcasing familial tenacity leads the way for perfect storytelling.
If you haven’t seen this film, seek it out at this website, follow Lainey Moseley for viewing opportunities. I’m so impressed with how well the documentary captures the human element and highlights scientific advancements, featuring genetic diagnosis and modern gene and cell therapy.
Steven Gruber
Read more at: linkedin.com
Our
Mission
Rare diseases affect more people than Cancer and AIDS combined.
There are over 10,000 rare diseases in the world. 1 in 10 people worldwide have a rare disease and 50% of these rare diseases affect children. But unfortunately, there are treatments for less than 5% of those diseases. As such, 30% of these children don't make it to their 5th birthday. And while no one thinks their child will be born with a rare disease, it can happen to anyone – nature does not discriminate.
Few can appreciate the battle families of kids with rare diseases fight. The mission of our film is to raise awareness about the diagnostic and cure odyssey of rare disease kids that falls primarily on the advocacy of parents. We ask the question, why isn’t more being done to save these kids? And if this was your child, what would you do?
When parents are told that their child has a rare genetic disease, they are advised that nothing can be done, that there are no treatments, and to “go home and enjoy your child.” But the science and success of gene and cell therapy is changing the landscape for rare diseases and giving families hope.
But finding a therapy and treating one child with a rare disease can cost up to 6 million dollars. And most pharmaceutical and Biotech companies are not incentivized because there is little to no profit margin developing drugs for rare diseases. So families must raise millions of dollars on their own and find scientists and Biotech companies willing to help save their child
We need to change the way we think about, talk about, research and cure rare diseases. We need to think of them in the aggregate, just like we think about over 200 diseases that we simply call “cancer.” The limits to research funding, the challenges to therapy development, the resistance to commercialization, in other words, our failure to save these children from these diseases stems from one characteristic - that they are rare.
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How can you support our rare disease families?
RARE tells the story of 6 rare disease families diagnostic and cure odysseys fighting for gene and cell therapies against the back drop of an indifferent healthcare system and a ticking clock.
Please email us about tax-deductible donations
There are no approved treatment options for 95% of rare diseases. At present, approximately 50 new therapies are approved globally each year. At this rate, it would take more than 100 years to develop a single treatment for every rare disease estimated to exist worldwide. But the advocacy of families is pushing science to work harder, faster and smarter to scale new therapies and save more kids.
The treatment options for rare diseases kids are complicated and risky. But when a child is sick and the clock is ticking to find answers, the families we profile here are finding hope by exploring the use of FDA approved repurposed drugs, ASO (Antisense Oligonucleotide Technology) and Gene Therapy. All have proven successful in improving and saving kids’ lives. And while only 5 gene therapies have been approved to date by the FDA, many more are in clinical trials and pipelines awaiting approval.
The Science of Gene and Cell Therapy
Call to Action
“ I hope 10 years from now,
no other parent has to be told that their child has a rare disease and that nothing that can be done."
- Casey McPherson
Rare disease parent advocacy is forcing legislative, science and industry leaders to take notice. This documentary hopes to help raise awareness that rare disease in the aggregate is a health crisis that people need to care about NOW. Without changes to the cost structure, regulatory process and availability of scalable treatment options, rare disease families will have to continue to fight this battle on their own.
please join the Everylife Foundation to learn more how you can help advocate for rare disease kids. They are working tirelessly in Washington, Dc and every state to empower the rare disease patient community to advocate for impactful, science-driven legislation and policy that advances the equitable development of and access to lifesaving diagnoses, treatments, and cures. www.Everylifefoundation.org
The families we profile in this film are the pioneers on the frontline of this battle, demanding change and action to save their child and every child born tomorrow with a rare disease.
Universal Newborn Screening Advocacy
Adopting Whole Genome Universal Screening is as important as finding treatments for rare diseases. The technology is there to pinpoint even the most ultra rare gene mutations at birth and to intervene on behalf of many rare diseases that might not be identified until it is too late. Please contact Everylife Foundation to learn more https://everylifefoundation.org/newborn-screening-take-action/
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The Filmmakers
Lainey Moseley
Producer and Director
John Beder
Co-Director
Lainey Moseley is an Emmy® award winning journalist who has worked for CBS and NBC News for the past 30 years. She is also the founder of CHOPS Syndrome Global; an organization raising awareness for her daughter Leta’s rare disease CHOPS Syndrome.
John Beder is an Emmy® Award winning documentary director and producer. John’s latest project explored pediatric palliative care and was part of the the Oscar-winning New York Times Op-Doc series.