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Cornell Employee from Montour Falls to be First to Receive New Therapy for Rare Disorder

Cornell Employee from Montour Falls to be First to Receive New Therapy for Rare Disorder

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Team Led by Monkol Lek Advances Past Pre-IND Phase with DMD Gene Therapy

Team Led by Monkol Lek Advances Past Pre-IND Phase with DMD Gene Therapy

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Recent

Organization Looks To Help Families Impacted By Rare Diseases

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

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Hope on the Horizon for Branford Boy, 5, with debilitating, fatal disease

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.

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Team Led by Monkol Lek Advances Past Pre-IND Phase with DMD Gene Therapy

Duchenne Muscular Dystrophy (DMD) was first documented in the 1860s. More than a hundred years later, researchers discovered the genetic mutation underlying the progressive muscle degeneration that defines this disorder. Despite further studies into the mechanism of DMD, no effective treatment currently exists. Like many rare genetic diseases, DMD ultimately results in loss of quality of life and death. But thanks to advances in gene editing technology, that could soon change.

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