May 28, 2019
With over 7,000 rare conditions already identified and dozens more being discovered every day, it is estimated that rare diseases impact 1 in 12 Canadians, two-thirds of whom are children. But each disease affects only a handful of individuals, so understanding and medical expertise are limited and uneven across the country. Many of these conditions […]
May 24, 2019
from ir.ptcbio.com
– $545,000 awarded to help improve Duchenne muscular dystrophy diagnosis and screening across the globe –SOUTH PLAINFIELD, N.J., May 20, 2019 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the three funding recipients from PRIORITY, an annual program to support outstanding clinical research by the rare genetic disorders community. This year’s funding will accelerate innovative research projects to improve Duchenne […]
May 14, 2019
from Newswire.ca
Government of Canada consults on new approach to fast-track the review of treatments for serious, rare and debilitating diseases OTTAWA, May 13, 2019 /CNW/ – The Government of Canada is committed to helping all Canadians access affordable treatment options they need to help them improve and maintain their health. For many Canadians, managing the symptoms of rare or debilitating diseases can be […]
May 6, 2019
A protein called epidermal growth factor (EGF) can help preserve muscle strength and increase muscle regeneration in a mouse model of Duchenne muscular dystrophy (DMD), a finding that may pave the way for new treatment strategies for DMD, researchers said. The study, “EGFR-Aurka Signaling Rescues Polarity and Regeneration Defects in Dystrophin-Deficient Muscle Stem Cells by Increasing Asymmetric Divisions,” […]
April 29, 2019
from prnewswire.com
– Ambulatory Duchenne patients who are 5 years and older with a nonsense mutation can now access a treatment that targets the underlying cause – SOUTH PLAINFIELD, N.J., April 29, 2019 /PRNewswire/ — PTC Therapeutics, Inc. today announced that Translarna™ (ataluren) has been granted marketing approval from the Brazilian National Health Surveillance Agency (ANVISA) under rare diseases […]
April 18, 2019
from Xconomy
If a once-modest regional hospital and its new biotech allies have their way, the capital of Ohio could one day rival America’s other biomedical hubs. “Our goal is to make Columbus the center of the universe for gene therapy,” says Doug Ingram, CEO of Sarepta Therapeutics. Sarepta (NASDAQ: SRPT) is based in Cambridge, MA, as you […]
April 10, 2019
from Muscular Dystrophy News Today
Using a genetic engineering tool to restore dystrophin while raising levels of utrophin, a similar protein, leads to better improvements in muscle function than either approach alone, research in a mouse model of Duchenne muscular dystrophy (DMD) reports. The study, “The potential of utrophin and dystrophin combination therapies for Duchenne muscular dystrophy,” appeared in the journal Human Molecular […]
April 5, 2019
Toronto, Ontario – Muscular Dystrophy Canada (MDC) and The Foundation for Gene & Cell Therapy (Jesse’s Journey) are joining forces to accelerate ground-breaking research focused on new treatments for Duchenne muscular dystrophy to the sum of $600,000. Through this partnership, MDC will provide $300,000 with Jesse’s Journey matching the commitment. Two research projects will be […]
April 3, 2019
from New York Times
Scientists are testing nearly two dozen treatments that might stop the disease. But enrollment in the trials is very restricted, and few children qualify. Lucas was 5 before his parents, Bill and Marci Barton of Grand Haven, Mich., finally got an explanation for his difficulties standing up or climbing stairs. The diagnosis: muscular dystrophy. Mr. […]
April 3, 2019
from Muscular Dystrophy News Today
New nine-month data on four boys with Duchenne muscular dystrophy (DMD) enrolled in Study-101 testing Sarepta Therapeutics‘ micro-dystrophin gene therapy continues to show “very encouraging” results, company officials said. These updated data, given in a recent investors’ webinar, show the gene therapy resulted in 81.2% increase in dystrophin protein expression in the muscles, accompanied by marked improvements in boys’ […]
