Pharma-sponsored rare disease film festival doubles in size, readies for West Coast debut

Rare Disease Film Festival image
The rare disease film festival Disorder goes to the West Coast this fall with 50 films, including one from founder Daniel DeFabio featuring his son who has Menkes Disease. (DeFabio)

Disorder: The Rare Disease Film Festival is back, and it's bigger than ever. Two years ago, the festival launched with 24 films. This year the biennial fest will screen 50 films over two days in November.

Also back are its pharma sponsors. Sanofi Genzyme, Horizon, CSL Bering, Vertex, Biomarin and Takeda, all makers of rare disease treatments, are helping foot the bill.

Takeda is additionally sponsoring a sneak preview of six of the films during Biotech Week Boston which are set to air at the Takeda Auditorium on September 12.

“While individual rare diseases are rare, taken collectively there are approximately 350 million people affected by rare diseases around the world. And without an approved treatment for the vast majority of these diseases, we are committed to not only working to develop new treatments but to also working with our partners to raise awareness of rare diseases,” Ramona Sequeira, president of Takeda's U.S. business unit, said via email.

She added that the Boston preview and the film festival help do that by bringing together different stakeholders to find ways to work together for patients.

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Another change in the festival is the venue. Disorder this year this will be in San Francisco, moving from Cambridge, Massachusetts, with the purposeful intent of opening doors to new audiences. Disorder co-founder Daniel DeFabio said he’s looking forward to introducing the films and stories to “a whole new cohort” of people.

“The great thing that we see happen is when we bring together the different stakeholders—of course the patient community and the advocates for patients—but also industry. They don’t just participate as sponsors, they actually attend and sometimes are among our speakers. And then clinicians and geneticists who might do the diagnosis. Getting all those same people in the same room to hear these stories, that is the goal. We hope it’s creating opportunities where people talk to each other and maybe start to think about a disease they weren’t focused on previously,” he said.

RELATED: Takeda, Microsoft rare disease partnership publishes diagnosis road map

DeFabio, whose son has a rare genetic disorder called Menkes disease, founded the festival along with Bo Bigelow, whose daughter has a rare USP7 genetic mutation. Both of their films, “Menkes Disease: Finding Help & Hope” and “Tess is Not Alone” are among the 14 that aired in the original festival that will now go to the West Coast.

“We put out the call for stories as ‘7,000 Films Wanted’ because ideally someday we’ll have 7,000 films representing all 7,000 rare diseases,” DeFabio said.

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