Since two gene therapies were approved for sickle cell disease in late 2023, only a handful of patients have started the ...

In the 1990s, European researchers developed a gene therapy cure for SCID-X1 and were able to report that children with the condition had been successfully treated. However, several years later ...

Even so, Jacobsohn acknowledged few patients have begun the gene therapy treatment. At Children ... told NBC News they have yet to see an “ultimate denial” for their drugs.

Genprex (GNPX) announced that the first patient has been enrolled and dosed in the Phase 2 expansion portion of the company’s Acclaim-3 ...

CHOP doctors Thompson and Grupp have devoted their lives to improving the quality and length of life for children with sickle ...

Solid Biosciences (SLDB) announced that it has received Fast Track designation from the U.S. Food and Drug Administration, FDA, for SGT-212, ...

The treatment offers a one-time gene therapy for patients with vision loss due to confirmed biallelic RPE65 mutation-associated inherited retinal disease (IRD).

An in vivo base editing approach targeting the PRNP gene led to a 52% increase in the lifespan of mouse models inoculated with the most common sporadic and genetic types of human pathogenic prion ...

Novotech, the global full-service clinical Contract Research Organization (CRO) that partners with biotech and small to mid-size pharma companies to accelerate the development of advanced and novel ...

Therapy also contributes to healing in ways no other treatment can. Because of the way the human nervous system is built, the presence of the therapist has a physiological effect that calms.

Psychotherapy, also called talk therapy or usually just "therapy," is a form of treatment aimed at relieving emotional distress and mental health problems. Provided by any of a variety of trained ...

Cystic fibrosis is caused by mutations in the gene that produces the cystic fibrosis transmembrane conductance regulator (CFTR) protein. In people with CF, mutations in the CFTR gene can disrupt the ...