BOULDER, Colo. — Edgewise Therapeutics, Inc., a leading muscle disease biopharmaceutical company, today announced the launch of an educational website dedicated to the Becker muscular dystrophy (Becker) community: www.beckermusculardystrophy.com. This is the first website solely focused on providing Becker-specific resources to help individuals and caregivers better understand the disease, learn...
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In April 2020, as the world went into lockdown and laboratories shuttered, Elizabeth Berry-Kravis was on the move. Her team’s clinical trial of a potential treatment for fragile X syndrome — one of the most common inherited forms of intellectual disability and a leading genetic cause of autism — was nearly complete. But she and her...
SAN DIEGO – Endeavor BioMedicines, a clinical-stage precision medicine company targeting the core drivers of multiple terminal diseases including oncology and fibrosis, announced that the company has dosed its first patient in a Phase 2 trial of taladegib (ENV-101) to treat idiopathic pulmonary fibrosis (IPF). Taladegib is a small-molecule inhibitor...
PARIS – ENTEROME SA, a clinical stage biopharmaceutical company developing novel therapeutics based on its unique ability to de-code molecular interactions in the gut microbiome impacting human health, is pleased to announce the appointment of Anne Dagallier, PhD, as Chief Business Officer (CBO). Dr Dagallier brings over 22 years of...
Brussels, Belgium – Multiple sclerosis (MS), an inflammatory disease of the central nervous system, affects more than 1 million people in Europe and an estimated 2.8 million worldwide. MS is characterized by an inflammation-related loss and damage of the myelin layer in the brain and spinal cord, which insulates the...
She is living her life to the fullest, even though she can’t walk or talk. Twelve-year-old Breanna Leatherwood of Tuscaloosa is one of about 50 females in Alabama known to have Rett syndrome, a neuro- developmental disorder that affects a gene on the X chromosome and occurs almost exclusively in...
The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each worth $5,000, to cover a winning applicant’s...
NEWBURYPORT — Colton Fontaine was the picture of good health. A standout athlete during his days at Newburyport High, Fontaine went on to enjoy a successful college baseball career at Salem State and now serves as the program’s top assistant coach. But that all changed in the blink of an...
CHICAGO & CAMBRIDGE, Mass. – Exicure, Inc.® (NASDAQ: XCUR), a pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA™) technology, today announced the appointment of Brian C. Bock as the Company’s Chief Financial Officer (CFO) effective immediately. “Exicure’s proprietary SNA platform technology holds great potential for treating...
CHICAGO & CAMBRIDGE, Mass. – Exicure, Inc. (NASDAQ: XCUR), a pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA™) technology, today announced the appointments of Elizabeth (Betsy) Garofalo, M.D. and Mr. Andrew Sassine to its Board of Directors. “We are pleased to welcome Betsy and Andrew to...