Repurposed FDA-approved drug could uncover potential treatment for rare genetic disorders
In a new study, researchers at McMaster University have identified a potential treatment for Sandhoff and Tay-Sachs ...
Nipocalimab demonstrates significant clinical improvement in disease activity and IgG reduction in Phase 2 Sjögren's disease study
Johnson & Johnson (NYSE: JNJ) today announced results from additional analyses of the Phase 2 DAHLIAS study highlighting improvement in key measures of disease activity and significant IgG reduction ...
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RNA Stock Hits Record High on Entering the Cardiac Disease Space
The company has decided to advance two new wholly-owned pipeline drugs, AOC 1086 and AOC 1072, targeting two rare genetic ...
BioSpace2h
PTC Wins First FDA Nod for Direct-to-Brain Gene Therapy, Targets Ultrarare Disease
The FDA has followed in the footsteps of its European counterparts and granted accelerated approval to PTC Therapeutics’ gene ...
Dallas Observer2h
Dallas Steakhouse Legend, Al Biernat, Dies at 69
On Wednesday, The Dallas Morning News reported that Dallas restauranteur Al Biernat died at 69 after a battle with ...
The Week2h
Tackling Spinal Muscular Atrophy: A long road ahead for India
Alpana Sharma, founder-director of CureSMA, a parent-led advocacy group, urges the government to include medicines for SMA in ...
ThePrint8h
Medicover Hospitals Successfully Performs Marathwada’s First Rare and Complex Simultaneous Liver-Kidney Transplant (SLKT)
Chhatrapati Sambhajinagar (Maharashtra) [India], November 14: Medicover Hospitals has marked a significant milestone in healthcare by successfully performing First time in Marathwada a rare and ...
Haryana: 14-Month-Old Kenyan Infant Successfully Treated For Rare, Life-Threatening Disorder, Pearson Syndrome, In Gurugram
Gurugram: Doctors at a private hospital in Haryana's Gurugram have successfully treated a 14-month-old baby from Kenya who was suffering from the extremely rare and life-threatening Pearson Syndrome.
ENDPOINTS NEWS10h
FDA ap­proves PTC’s ul­tra-rare dis­ease gene ther­a­py for broad­er pa­tient pop­u­la­tion than Eu­rope
The FDA has cleared PTC Ther­a­peu­tics’ gene ther­a­py for AADC de­fi­cien­cy, an ul­tra-rare dis­ease that im­pacts the ...
YTN10h
Establishment of a basis for supporting the production and seller of medical devices for rare diseases
but the scope of support has expanded as the "Rare Disease Control Act amendment" passed the National Assembly. Ji Young-mi, ...
AstraZeneca Q2 net profit declines 10%, revenue up 31%
AstraZeneca’s growth was driven by its portfolio of products in the oncology, biopharmaceuticals and rare disease segments ...