Patient-informed discovery and patient-driven development
Patients are central to our mission at Fulcrum. Partnering with the patient community is also essential to our process. Our drug discovery begins with a deep exploration of disease biology in patient tissue samples. As we advance a program, the patient voice guides our development strategy.
Our initial areas of focus are facioscapulohumeral muscular dystrophy (FSHD), sickle cell disease (SCD), and non-SCD hemoglobinopathies, including beta-thalassemia. We’re developing precision medicines that address the root causes of these genetic conditions.
Inspired by patients
At Fulcrum, we are driven by a commitment to make a difference for patients with genetically defined diseases. We have been privileged to spend time with many of these patients and their families. Their experiences guide us as we work to develop therapies that will lead to meaningful improvements in their day-to-day lives. Read more about the patients who inspire us.
FSHD
Lexi
Boston, Massachusetts | Born January 1994
“Anything I can do, I will do. Even if it’s hard.”
Growing up, Lexi watched her mother, maternal uncle and grandfather struggle and overcome the challenges of living with facioscapulohumeral muscular dystrophy (FSHD), unaware that she, too, had the genetic mutation that causes the condition. In high school, a memoir writing class inspired her to create a documentary telling the story of FSHD in her family – a film which now has over 40,000 views.
Sickle Cell Disease
Kadeem
Boston, Massachusetts | Born May 1989
“It’s hard for sickle cell patients to get their voices heard… We have an invisible disability. That’s what my poetry aims to express.”
Kadeem has spent his life navigating between periods of normal functioning and periods of hospitalization, seclusion and anxiety due to sickle cell disease (SCD). As a teenager, he began to let his mind play with words, rhythms and sounds. Over the years, spreading awareness about SCD through poetry and advocacy has become Kadeem’s medicine of choice.
FSHD
Shawn
Orange, Connecticut | Born February 1959
“I don’t want any kid who is disabled to ever think that they are less than anyone else.”
Cars are the love of Shawn’s life – nothing supersedes the pure, uninhibited enjoyment he gets from driving. Facioscapulohumeral muscular dystrophy (FSHD) has forever altered Shawn and his family’s landscape, his goals and aspirations and the cars he gets to drive. However, he refuses to let the disease define his personality.
FSHD
Katelyn
Boston, Massachusetts | Born January 2011
“My life is 99% happy and 1% frustration. And that frustration is FSHD.”
Katelyn first began to show signs of facioscapulohumeral muscular dystrophy (FSHD) when she was six years old. Still, she approaches life with confidence – she is frank, poised and self-reliant, largely unfazed by other peoples’ opinions. That positivity is matched by her mother’s motivation to help find a therapy for FSHD and to bolster the FSHD community.
Sickle Cell Disease
Teonna
Baltimore, Maryland | Born July 1991
“I have a lot of hope. In spite of everything, I am still able to smile.”
Teonna was diagnosed with sickle cell disease (SCD) as an infant, following a routine screening at the pediatrician’s office. As a young person, she hid her pain from those around her out of fear of being different. Today, she is an advocate for the SCD community – her way of honoring those who have passed and working for better treatment options for her fellow sickle cell warriors.
