Your muscles need continuous maintenance to stay strong and healthy. Duchenne muscular dystrophy (DMD) happens when there is a change in a person's genetic instructions that affects the production of ...
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that on ...
"The incidence of DMD in our study was similar to that reported in previous studies . . . Overall, our results indicate that newborn screening for DMD is feasible, and this will be good news for the ...
Endoxifen program into rare pediatric neuromuscular disease along with previously received Rare Pediatric Disease Designation SEATTLE, /PRNewswire/ -- Atossa Therapeutics, Inc. (Nasdaq: ATOS) ("Atossa ...
From imaging innovations to new treatment approvals, 2024 brought advancements in Duchenne muscular dystrophy care and research. This year’s top stories on Duchenne muscular dystrophy (DMD) managed ...
Investigators based in Italy recently assessed the impacts of swallowing difficulties in a cohort of patients with DMD who presented to a single hospital with respiratory issues. The Swallowing ...
SAN DIEGO, June 25, 2024 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare ...
FDA has requested the HOPE-3 clinical study report (CSR) as part of the BLA review processCompany expects to submit updates to the BLA in ...
Duchenne muscular dystrophy (DMD) is a genetic condition that causes increasing muscle weakness over time. While DMD currently has no cure, researchers are studying potential new treatments. When ...
Catalyst is offering patients and their caregivers a personalized program called Catalyst Pathways to provide guidance and financial support. Agamree ® (vamorolone) oral suspension has been made ...
Duchenne muscular dystrophy (DMD) is a muscle degeneration disorder caused by mutations affecting the dystrophin gene. Researchers show how a dual CRISPR RNA method restored dystrophin protein ...
The primary endpoint and key secondary endpoints were met for the vamorolone 6mg/kg/day treatment group. The Food and Drug Administration (FDA) has approved Agamree ® (vamorolone) for the treatment of ...
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