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RADNOR, Pa. – Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders, today announced that due to the impact of the COVID-19 Omicron variant on hospital resources and an unexpected interruption of clinical supply material associated with IV ganaxolone, the... -
Over 90 percent of patients with transfusion-dependent thalassemia, an inherited blood disorder, no longer needed monthly blood transfusions years after receiving gene therapy, according to an international Phase 3 clinical trial that for the first time included children younger than 12 years of age. Twenty-two patients were evaluated (ranging in...
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City of Hope, one of the largest cancer research and treatment organizations in the United States and a leading research center for diabetes and other life-threatening illnesses, today announced that its Department of Stem Cell Biology and Regenerative Medicine received a $4.9 million grant to train the next generation of...
Videos
Rare disease spotlight
Watch David's film now: https://www.hda.org.uk/huntingtonsinmind
@HDA_tweeting are sharing three films to raise awareness of #HuntingtonsDisease. They highlight real stories of those affected by the condition.
Keep an eye out for the release of final film
#HuntingtonsInMind #RareDisease https://twitter.com/HDA_tweeting/status/1526558041243635713
Watch David's film now live on our website. Both David and Charlie's films are now available to watch and both inspiring stories highlight some of the #MentalHealth challenges associated with #HuntingtonsDisease.
#HuntingtonsInMind
Watch both films now:
https://loom.ly/GBFyPOc
An est. 16.8M people in the U.S. are caring for a child with disabilities or complex medical needs. The #Unseen documentary takes a raw, honest look at the parent caregiving experience. Watch the trailer & more👇👇
https://caregiverdoc.com/
#LaDDC
#FeatureFriday
#CaregiverDoc