Although rare diseases are individually rare, around the globe nearly 400 million people suffer from approximately 7,000 different rare diseases. Approximately 80% of all rare diseases are genetic in ...

Steven Pittler, Ph.D., at the University of Alabama at Birmingham has sought to find modifier genes for the hereditary eye disorder retinitis pigmentosa type 59. After onset in one’s late teens, RP59 ...

Please provide your email address to receive an email when new articles are posted on . Low-dose OCU410 slowed lesion growth, preserved retinal tissue and stabilized visual function in three patients.

Panelists discuss how splice modifiers work by enhancing protein production from the SMN2 gene, with risdiplam (Evrysdi) being an oral daily medication and nusinersen (Spinraza) being administered ...

MALVERN, Pa., Aug. 05, 2024 (GLOBE NEWSWIRE) -- Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and ...

Non-compaction cardiomyopathy is a heart condition caused by defects that arise during fetal development and can have diverse health impacts in affected individuals, including sudden cardiac death.

MALVERN, Pa., June 05, 2025 (GLOBE NEWSWIRE) -- Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced the ...

MALVERN, Pa., May 27, 2025 (GLOBE NEWSWIRE) -- Ocugen, Inc. (OCGN) (Ocugen or the Company), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that the United ...

CHICAGO (Reuters) - Scientists have discovered a genetic mutation linked with colon cancer that may work like a spigot, controlling the number of precancerous growths that develop and determining a ...

BIRMINGHAM, Ala. – Modifier genes add complexity to precision medicine. A modifier gene is one that has genetic variants such as single nucleotide changes that differ from the most common sequence in ...