Episode 72: Trying to Outpace Rare Disease
Rich Horgan, founder and president of Cure Rare Disease(CRD), discusses how he sprang into action when his brother was ...
Tag: rare
Top 5 Most-Read Rare Disease Stories of 2020
The top 5 most-read stories on rare diseases of 2020 on AJMC.com focused mainly on new and noteworthy treatment ...
Santa makes special visit to Wilmington girls suffering from rare disease
John joined the WECT sports team in January of 2010 after spending the past six years in Bangor, Maine. ...
UF basketball player diagnosed with rare heart disease after court collapse
GAINESVILLE An elite athlete in Florida was diagnosed with a rare heart disease after collapsing during a UF ...
Chiesi Global Rare Diseases Announces First Patient Treated in Expanded Access Program for Pegunigalsidase Alfa for Proposed Treatment of Fabry Disease
BOSTON, Dec. 23, 2020 /PRNewswire/ -- Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A., an international research-focused ...
Covid-19: Heart disease avoidable deaths caused due to rare hospital visit – health
A new study led by UCL researchers has found out that during the initial phase of the Covid-19 pandemic, ...
New drug molecules hold promise for treating rare inherited terminal childhood disease
Scientists at the University of Exeter have identified a way to "rescue" cells that have genetically mutated, paving the ...
Polaryx Therapeutics Receives Both Rare Pediatric Disease and Orphan Drug Designations for the Treatment of Niemann Pick Disease Types A and B With PLX-300
PARAMUS, N.J., Dec. 18, 2020 /PRNewswire/ -- Polaryx Therapeutics, Inc., a biotech company developing small molecule therapeutics for lysosomal storage disorders, announced today that it ...
Polaryx Therapeutics Receives Both Rare Pediatric Disease and Orphan Drug Designations for the Treatment of Niemann Pick Disease Types A and B With PLX-300
BenzingaWatch Out, Elon Musk. These EV Startups Are Trying To Take On TeslaWith a $145.9 billion fortune at press ...
Community supports coach battling rare disease
GRAND MEADOW, Minn. - A high school baseball coach and former RCTC football player is facing a new challenge. ...
