The American Journal of Managed Care1h
Prophylactic Cardiac Medication May Prolong Survival in DMD
Prophylactic cardiac treatment may prolong survival in Duchenne muscular dystrophy (DMD), but only one-quarter of individuals ...
Lincoln Journal Star4h
Lincoln gets $600,000 federal grant for inclusive playground at Antelope Park
The city City got a $600,000 federal grant to help pay for a $2 million renovation of the Antelope Park playground into a ...
Personalized therapy for rare genetic diseases: Patient-derived organoids offer new hope
Children's Mercy Kansas City has achieved a significant advancement toward the treatment of rare genetic diseases through the ...
Muscular Dystrophy News7h
What success looks like for me as a DMD parent
As a parent and caregiver to three sons with DMD, columnist Betty Vertin's definition of what success looks like has changed over the years.
precisionmedicineonline7h
Belief BioMed's Duchenne Muscular Dystrophy Gene Therapy Gets FDA IND Clearance
The Shanghai-based firm will test the gene therapy candidate in patients between 4 and 9 years old with Duchenne muscular dystrophy.
The Pharma Letter12h
RPD designation for Nippon Shinyaku’s DMD candidate
The US Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation to NS-051/NCNP-04, which is ...
Gene therapies have been uneven for DMD — but these companies hope to turn the tide
Despite mixed results using gene therapies to treat Duchenne muscular dystrophy, drug developers are pushing ahead with the ...
Giving back to the people, West Texas Rehab Telethon event
The West Texas Rehab Telethon will be taking place this weekend. This event is to give back to people and facilities that care for people in need.
What is William Blair’s Estimate for SRPT FY2024 Earnings?
Analysts at William Blair boosted their FY2024 earnings per share estimates for shares of Sarepta Therapeutics in a research ...