New Analysis of Boehringer Ingelheim’s IPF Therapy Nintedanib on Lung Function Decline


A pre-specified, pooled subgroup sensitivity analysis from the two identically designed Phase III INPULSIS™ trials, presented today at the European Respiratory Society International Congress (ERS) evaluated the impact of the investigational drug nintedanib on reducing the decline in lung function, as measured by annual rate of decline in forced vital capacity (FVC), in patients with idiopathic … [more here...]

Edison Pharmaceuticals Holds Out Hope for Failed Rett Syndrome Drug Trial

Guy Miller, MD, PhD, CEO of Edison Pharmaceuticals, Inc

An Edison Pharmaceuticals Inc. drug focused on a neurological disorder in young girls failed a mid-stage trial, but the Mountain View company still … [more here...]

Platinum Recording Artist and American Idol Season 8 Winner, Kris Allen, Performs At Global Genes’ “Tribute to Champions of Hope” Gala Access Hollywood LIVE co-host Kit Hoover, along with Access Hollywood co-host Liz Hernandez, actively participate


It is estimated that it takes an average of five to seven years for a patient suffering from a rare disease to receive a proper diagnosis, with 95% of … [more here...]

Opexa Therapeutics to Target Rare Disease Neuromyelitis Optica (NMO) as next Development Program

Neil Warma, CEO of Opexa Therapeutics Inc.

Opexa Therapeutics, Inc. (NASDAQ: OPXA), a biotechnology company developing Tcelna® (imilecleucel-T), a novel T-cell immunotherapy for the treatment … [more here...]

The Rare Daily: RARE Facts in Under a Minute

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Attended the 2014 Global Genes Summit and Gala? Share Your Story

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Each year Global Genes puts together a two day Patient Advocacy Summit and our Tribute to Champions of Hope Gala. For the Global Genes team it means … [more here...]

Photo & Video Roundup of 2014 Global Genes Summit and Gala Events

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We would like to extend a very heartfelt THANK YOU to everyone who spent the last few days with us online and in-person at the 3rd Annual RARE Patient … [more here...]