During the past decade, Parkinson disease (PD ... of different forms of monogenic PD has revealed an unexpectedly large amount of clinical and genetic heterogeneity. Additional complexity is ...

Over the past 15 years, breakthroughs in basic science have revealed the genetic etiology for several inherited cardiovascular diseases. [1,2] By studying instructive monogenic diseases ...

Immunosuppressants used in adeno-associated virus (AAV) gene therapy for monogenic disorders present an adverse effect profile that is consistent with high-dose steroid use in other conditions ...

Little research has explored family experiences after preimplantation genetic testing (PGT) for monogenic disorders (PGT-M) ...

Dyno Therapeutics has recently announced the formation of a new strategic partnership with Swiss pharma giant Roche to ...

With the formation of a new strategic partnership, Dyno Therapeutics is responsible for the development of novel AAV capsids.

Genetic testing in the diagnosis of chronic ... Clinical impact of genomic testing in patients with suspected monogenic kidney disease. Genet Med 2021; 23: 183–91 CrossRef 15.

Cantor Fitzgerald analyst Kristen Kluska maintained a Buy rating on Taysha Gene Therapies (TSHA – Research Report) yesterday and set a ...

In this case report, Gad and colleagues performed corneal confocal microscopy to assess for evidence of nerve regeneration ...

London – 28 October 2024 - Purespring Therapeutics, a pioneering gene therapy company focused on transforming the treatment of kidney diseases, presented preclinical data at the American Society of ...