$500,000 initiative with Toronto children’s hospital aims to develop genome editing therapies for Duchenne muscular dystrophy patients

Researchers and patients are excited about recent advancements, but such experiments have their own sets of risks and challenges

On Dec. 5, 2003, Ian Sharp was diagnosed with Becker muscular dystrophy. He was only 10 years old. From that day on, the Sharps, a local Aspen family, have been on a desperate search to find a cure for their son.

NEW HAVEN, Conn. — Terry Horgan is a 26-year-old who works at Cornell University. He’s very tech savvy. Currently, he gets around in a motorized wheelchair due to a rare disease called Duchenne muscular dystrophy.Linda Horgan is Terry’s mother.“When we found out Terry had this, it’s like life left you," Linda said. "It’s like being in an elevator and it just drops.”Muscular dystrophy is characterized by the progression of muscle weakness throughout one’s life....

Terry Horgan, a Cornell graduate and now a Cornell employee, will be the first person in the world to undergo a possible cure for Duchenne muscular dystrophy. Horgan's brother Rich, also a Cornell graduate, is the founder and president of Cure Rare Diseases, the biotech nonprofit behind the treatment.

BRANFORD — Max Herzog, 51/2, was a “strapping boy” of almost 11 pounds at birth, so it was a shock for his parents to hear at about 6 months old that he had Duchenne muscular dystrophy, a rare disease that eventually would put him a wheelchair, then take his life.

Cure Rare Disease, a nonprofit biotechnology research organization, will hold a fundraising event on June 5 in Stamford.

Richard Horgan – 29-year-old Richard Horgan is the founder of Boston-based Cure Rare Disease, a non-profit biotech that develops custom-made drugs for rare diseases, including Duchenne muscular dystrophy. Horgan’s brother Terry is a DMD patient. Cure Rare Disease is currently working on a CRISPR-based therapeutic for this disease. The non-profit has raised about $2.3 million to conduct research on this drug, as well as others in its pipeline.

Duchenne Muscular Dystrophy (DMD) was first documented in the 1860s. More than a hundred years later, researchers discovered...

Ep269: Changing the World One Step at a Time, with Cure Rare Disease founder Rich Horgan

The gaming community is getting behind a Boston biotech entrepreneur’s quest to cure his younger brother’s rare genetic muscle condition. For the better part of his adult life, Rich Horgan has been working to fund a customized gene therapy designed specifically for his brother, Terry Horgan, who suffers from Duchenne muscular dystrophy, a rare inherited disease that causes significant muscle degeneration over time — and one that is fatal.

For many rare disease patients, the wait for treatment and research advancements can feel like a never-ending process. This wait is made…

Recognizing the Nonprofit Biotech Founder’s Leadership in the Groundbreaking Development of Customized Therapeutics to Treat Duchenne Muscular Dystrophy & Other Rare Diseases...

As the US learned to handle surges in COVID-19 cases that overwhelmed hospitals and shut down cities and confronted systemic racism amid protests sparked by the police killing of George Floyd, healthcare leaders have been navigating a new reality.‍

An organization devoted to offering treatment to those diagnosed with rare genetic disorders, Cure Rare Disease (CRD), has announced a…

Leadership, to me, is defined as having the courage to imagine a better future and the conviction to not give up despite the challenge. No challenge worth solving is easy and it is a leader’s job to convince others to believe in the vision of a better tomorrow. Whether this is solving the problem of […]

Terry Horgan, 24, is suffering from a rare form of muscular dystrophy. His brother Richard created a nonprofit called Cure Rare Disease...

Rich Horgan’s younger brother, Terry Horgan, is the inspiration behind Cure Rare Disease...

Rich Horgan has assembled a team to quickly develop a gene-editing therapy for his brother’s rare form of muscular dystrophy. Other people with rare diseases hope they are next

A custom antisense oligonucleotide drug has set records for both personalization and speed in drug development