Whether you turn red when drinking alcohol, dislike certain smells, or metabolize drugs differently from others, the ...
In the pivotal phase of the CAMPSIITE trial (n=13), participants through one year sustained an 82% median reduction of cerebrospinal fluid (CSF) levels of heparan sulfate (HS) D2S6, a key biomarker of ...
On Friday, REGENXBIO Inc. (NASDAQ:RGNX) shared new data from the Phase 1/2/3 CAMPSIITE trial of clemidsogene lanparvovec (RGX-121) for the treatment of patients with Mucopolysaccharidosis Type II (MPS ...
The U.S. Food and Drug Administration (FDA) on Monday extended its review timeline for REGENXBIO Inc.’s (NASDAQ:RGNX) Biologics License Application (BLA) for clemidsogene lanparvovec (RGX-121) for ...
Sarepta Therapeutics is still assessing how best to prevent liver injury from its commercialized Duchenne muscular dystrophy gene therapy. But Sarepta partner Hansa Biopharma has encouraging ...
Beacon Therapeutics may have found a guiding light in a disease area where other companies have stumbled with its laruparetigene zovaparvovec (laru-zova), a gene therapy that proved early improvements ...
A pair of clinical data sets gives Novartis confidence in a new formulation of the company’s gene therapy Zolgensma for the treatment of older patients with spinal muscular atrophy (SMA). Among SMA ...
Two-year data showed significant functional improvements in patients treated with delandistrogene moxeparvovec compared to controls, despite initial trial endpoint failure. The treated group exhibited ...
Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...
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