The following rare disease films will be screened at Biotech Week Boston
A portion of the event proceeds will be donated to the Rare Outreach Coalition’s 7000 Films initiative, which matches rare disease communities with a filmmaker and funding to tell the rare disease story through a film.
A story about the life of Lou, who is diagnosed with Angelman syndrome. Filmmaker Mika Matin stopped by the Bichell household for about an hour, with no advance planning, and no script, and captured everyday moments with Lou.
Directed by Mika Matin
Menkes Disease: Finding Help and Hope
Academy Award nominated actress Mary McDonnell narrates this look at a rare fatal genetic disorder called Menkes Disease. It prevents boys from metabolizing copper. Boys who get treatment in the first ten days of life can have long and relatively normally lives. If the disease is not detected and treated that early their lives are much shorter and far from normal. This short documentary aims to explain some of the basics of the disease and treatment but also provide context with examples from three families across the globe showing that there is life after this terrible diagnosis. And that life is more joyful and hopeful than you might expect.
Directed by Daniel DeFabio
Unconditional: Raising a Glass Child with a PACS1 Sibling
The phrase glass child refers to a sibling who might be neglected or given less attention because their brother or sister has more intense or dramatic needs. This is not only due to rare disease, but that’s a striking example. Unconditional is the first film created as a result of the 7,000 Films Wanted initiative.
Directed by Jon Dorflinger
A 3D computer-animated short, a CGI depiction of a boy in wheelchair getting left out of schoolyard fun and what it takes to include him.
Directed by Abel Goldfarb
Harry was born with a rare cranio-facial condition called Goldenhar syndrome which means that some of his features never developed. His twin brother was unaffected by the syndrome. People react to Harry in various ways. Some smile. Others whisper, stare, point and laugh. Online, people have said some truly awful things about the way he looks. This film sends a message of understanding and inclusion helping people realize the power in seeing each other for who they are and not just what they look like.
Directed by Hanna Brochocki, Ben Allen, Claire Banks, Ryan McCann, and Lauryn Mellor
Tess is Not Alone: A USP7 Story
When the parents of 7-year-old Tess Bigelow learned of her ultra-rare genetic disorder, they believed she was the only one of her kind. This short documentary tells the story of their worldwide quest to find others like Tess.
Directed by Bo Bigelow
USP7 Gene Mutation
Edge of Hope
A film about rare disease and the heroes going to the ends of the earth to create hope where none existed. Follow the bold adventures of a father climbing mountains for his dying boys, a woman running marathons blindfolded in honor of her sister, and a father biking solo across the country and back to raise awareness for the disease that stole his 1-year-old son.
Directed by Dan Salvo
Batten Disease, Sanfilippo Syndrome, Epidermolysis Bullosa, Myfio Myopathy
Counting Every Second
This is the story of Hannah Sames. A beautiful, young girl with a courageous spirit battles her only enemy—the ticking clock. What happens when the powerful will of a loving family intersects with people that dedicate their lives to scientific discovery?
Directed by Andrew Puccio
Giant Axonal Neuropathy (GAN)