Dogs always have been known as man’s best friend, but when it comes to seizure-response dogs for those with epilepsy, these special canines become invaluable protectors. The Walker family in north suburban Grayslake — mom Candace and children Colin, 11, Carson, 9, and Cailean, 5 — know this all too...
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POWAY — A Poway man afflicted with a life-threatening skin disease has passed a major obstacle on his way to an experimental stem-cell treatment. Mark Buenconsejo, 51, already has raised money with fundraisers and has successfully fought an insurance company that originally had refused to cover the expenses for a...
Mainz, Germany – The protein TDP-43 is present in all cells of our body and important for their biochemical processes. However, this protein can aggregate into large clumps in the brain, which can cause degenerative diseases such as Alzheimer’s and other dementias. How exactly this happens and how these protein...
PARIS, France – In a groundbreaking endeavour set to span five years, the DREAMS consortium is paving the way for a novel approach to treating neuromuscular diseases. This transformative project merges the power of artificial intelligence, pluripotent stem cells, and cutting-edge pharmacological screening techniques to seek out therapeutic solutions for...
DURHAM, N.C. — Glen Jernigan will meet his bone marrow donor for the first time on June 20 at the Duke Adult Bone Marrow and Stem Cell Transplant Program’s Patient Reunion. In 2005, Jernigan was diagnosed with aplastic anemia and recalls having more than a hundred blood transfusions before receiving...
STRASBOURG, France and PHILADELPHIA – Dynacure, a clinical-stage company focused on developing and commercializing novel therapies to transform the lives of patients with rare diseases who have limited or no treatment options, today announced its recognition of Rare Disease Day 2021. Dynacure is helping to raise awareness of myotubular and...
Dystonia sounds more like an Eastern European country than a health disorder. And like so many other diseases, conditions and disorders, the average person has probably never heard of it. It is only when dystonia affects a friend or family member that people start to ask questions. What is dystonia?...
BOULDER, Colo. – Edgewise Therapeutics, Inc., (NASDAQ: EWTX), a clinical-stage biopharmaceutical company focused on developing orally bioavailable, small molecule therapies for rare muscle disorders, today announced the appointment of Joanne M. Donovan, M.D., Ph.D., to the position of Chief Medical Officer. Dr. Donovan will be responsible for leading clinical, medical...
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...
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...