Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy slowed disease progression and showed sustained benefit ...
EPIDYS trial met primary endpoint demonstrating statistically and clinically meaningful treatment benefit in one of the largest DMD phase 3 trials to date Median follow-up of over 3 additional years ...
When it comes to children born with rare diseases, early diagnosis can make the difference between life and death. Since many ...
The Brain Involvement iN Dystrophinopathies (BIND) screener is a robust tool that facilitates early identification of ...
In research published in Developmental Medicine & Child Neurology, investigators have developed a brief, reliable, and valid ...
Precision BioSciences, Inc. , a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to ...
Dec 16 (Reuters) - U.S. Health Secretary Robert F. Kennedy Jr. on Tuesday added two rare genetic disorders, Duchenne Muscular Dystrophy and Metachromatic Leukodystrophy, to the federal newborn ...
The Enquirer and United Way of Greater Cincinnati have joined forces for the 39th year to help families in need with the Wish List program. After wishes are granted, remaining funds assist people with ...
Capricor Therapeutics reported positive data from its phase three study of its Deramiocel therapy to treat Duchenne muscular dystrophy. The company said the study met the primary endpoint and the key ...
UPSHER-SMITH LAUNCHES KYMBEE™ (DEFLAZACORT) TABLETS FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY
Families are encouraged to ask your doctor about KYMBEE™ (deflazacort) Tablets. If you or your doctor have questions about KYMBEE™, reach out to Upsher-Smith at 1-888-650-3789. Do not take if you have ...
EARLY ON, THE US POLO ASSN PALM BEACHES MARATHON IS LESS THAN A MONTH AWAY, AND ONE OF THE MANY ORGANIZATIONS RUNNING THIS YEAR IS FIREFIGHTERS ON A MISSION. IT’S A GROUP MADE UP OF PALM BEACH COUNTY ...
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