SAR446268 is a one-time adeno-associated viral gene therapy that delivers a vectorized RNA interference to silence DMPK expression. The Food and Drug Administration (FDA) has granted Fast Track ...
Congenital syphilis has a seen a dramatic rise since 2019, CDC data shows. The New York State Department of Health (NYDOH) is warning about the risks of congenital syphilis after three infant deaths ...
We present the case of a 31-year-old term parturient with a history of extensive thoracolumbar spinal fusion and a repaired anomalous origin of the left coronary artery from the pulmonary artery ...
(RTTNews) - argenx SE (ARGX) announced that it plans to advance ARGX-119—a first-in-class agonist antibody targeting muscle-specific kinase (MuSK)—to a registrational study in patients with congenital ...
Next-generation sequencing has enabled precision therapeutic approaches that have improved the lives of children with rare diseases. Congenital diarrhea and enteropathies (CODEs) are associated with ...
“Novocaine” is now in theaters, and its entire premise hinges on the idea that its hero simply cannot feel pain. Naturally, it begs the question: Is his genetic condition real? Directed by Dan Berk ...
Paramount Pictures has shared the final Novocaine trailer for its newest action comedy featuring The Boys star Jack Quaid as an ordinary man who has an extraordinary rare condition called congenital ...
If you thought The Boys was violent, get ready for Novocaine, an original new action movie starring Jack Quaid as a man with an extraordinary power: he can’t feel pain. You’ve probably been asked at ...
Documentary filmmaker Melody Gilbert has created a small gem of a film about a rare congenital disease known as congenital analgesia. Only about a hundred people worldwide are known to have this ...
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