Celebrating an Angelversary for Daughter with Trisomy


Our daughter, Mataya Grace, was diagnosed at 22 weeks gestation with Trisomy 13. We were told her defects were so severe that we should expect her to die before she was born. We made the decision to put her life in God's hands and to enjoy her for as long as He would allow. Mataya was born October 21, 2013 at 39 weeks  and three days. Not only was she born ALIVE and full term, she lived for … [more here...]

My Gala and Summit Experience: Carri Levy Creator of Behind the Mystery: Rare and Genetic

Carri Levy from Lifetime TV's Behind the Mystery: Rare and Genetic.

I met Nicole Boice, the founder of Global Genes over two and a half years ago through an interview for a new series that we were creating on rare … [more here...]

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 … [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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