Combining their individual fortés, the laboratories of Dave Holtzman at Washington University, St. Louis, and Brian Bacskai and Brad Hyman at Massachusetts General Hospital, Charlestown, treat us to ...

Please provide your email address to receive an email when new articles are posted on . Recessive dystrophic epidermolysis bullosa is a rare and serious condition. Zevaskyn is the first FDA-approved ...

Please provide your email address to receive an email when new articles are posted on . Krystal Biotech submitted a marketing authorization application to the European Medicines Agency for dystrophic ...

Recombinant adeno-associated viral vectors (rAAVs) are used for therapeutic gene transfer in skeletal muscle, but it is unclear if immune reactivity to gene transfer and persistence of transgene are ...

Credit: Krystal Biotech. Vyjuvek is a topical HSV-1 vector-based gene therapy. The Food and Drug Administration (FDA) has approved Vyjuvek ™ (beremagene geperpavec-svdt) for the treatment of wounds in ...

Prademagene zamikeracel (Zevaskyn) is the first and only cell-based gene therapy for wound treatment in patients with recessive dystrophic epidermolysis bullosa. The approval is based on the pivotal ...

The deficiencies noted in the CRL were not related to efficacy or safety data. The Food and Drug Administration (FDA) has issued a Complete Response Letter to Abeona Therapeutics regarding the ...

This story is part of a series on the current progression in Regenerative Medicine. This piece is part of a series dedicated to the eye. In 1999, I defined regenerative medicine as the collection of ...

The Food and Drug Administration on Friday approved the first treatment for a devastating condition that causes the skin to be so fragile that even a touch can cause it to splinter, bringing another ...

Focal adhesion (FA) proteins may be critical elements in the production of dystrophic neurites in Alzheimer's disease, according to research presented in the January 15 Journal of Neuroscience. Recent ...

A remote, downstream event in the pathology of muscular dystrophy may have a key role in the disease (pages 325–330). It seems that induction of nitric oxide synthase causes calcium to leak inside the ...

The study, published in Lancet Neurology, detailed the "head-to-head" trial implemented by the researchers to test two drugs, mexiletine and lamotrigine, on people with the condition. The trial, which ...