Muscular Dystrophy Association Announces 2025 MDA National Ambassador Lily Sander
The Muscular Dystrophy Association (MDA) has selected Lily Sander from Charlotte, North Carolina, as its 2025 MDA National ...
biopharma-reporter2d
Roche’s Elevidys demonstrates significant benefits for DMD patients in Phase 3
The gene therapy improved motor functions in children with Duchenne muscular dystrophy two years after treatment ...
appliedclinicaltrialsonline1d
Elevidys Shows Continued Promise in Phase III EMBARK Trial for the Treatment of Duchenne Muscular Dystrophy
In year two of the trial, Elevidys demonstrated statistically significant and clinically improvements across three key ...
BioSpace3d
Duchenne Muscular Dystrophy Space on Cusp of Pivotal Era
While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several ...
PMLiVE2d
Roche/Sarepta announce two-year results for Duchenne muscular dystrophy gene therapy
Roche and Sarepta Therapeutics have shared positive top-line results from a late-stage study of Elevidys (delandistrogene ...
manilatimes2d
MDA Launches 75th Anniversary Campaign and Premieres New PSA to Shine a Light on Rare Diseases
MDA launches 75th Anniversary campaign and premieres new public service announcement (PSA) to mark 75 years of impact in the ...
Sidra Medicine Establishes Gene Therapy Centre For Rare Paediatric Diseases
Sidra Medicine, a member of Qatar Foundation, has established a gene therapy centre to treat rare genetic diseases such as ...
ukauthority.com5d
Kent NHS trust runs pilot of AI for note taking
Kent Community Health NHS Foundation Trust (KCHFT) has begun a three-month pilot using the TORTUS AI assistant for taking ...
Qatar Tribune on MSN1d
Qatar: Sidra establishes Gene Therapy Center for rare genetic diseases
The announcement was made at Arab Health 2025 where Sidra Medicine will highlight its international patient service programmes ...
The Pharma Letter6d
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 ...
Kate Houck on leadership: ‘It’s no longer about you. It’s about empowering your people’
Executive director and ‘chief energy officer’ brings positivity, servant leader mentality to David’s Refuge, a nonprofit ...
Pharmabiz2d
Roche announces positive results from EMBARK phase III study of Elevidys in ambulatory individuals with DMD
Roche announces positive results from EMBARK phase III study of Elevidys in ambulatory individuals with DMD: Basel Tuesday, January 28, 2025, 11:00 Hrs [IST] Roche announced posit ...