Understanding the precise mechanisms of monogenic autoinflammatory diseases provides crucial insights into the complex pathophysiology of more common multifactorial diseases, while treatments ...

Financially, Rhythm has $319.1 million in cash, but may need to raise more funds within the next 12 months. See why I rate ...

LIPID inCode tests patients to identify familial hypercholesterolemia and prevent cardiovascular diseaseOXFORD, ENGLAND / ACCESSWIRE / September 20, 2024 / GENinCode Plc (AIM:GENI), the genetics ...

LIPID inCode tests patients to identify familial hypercholesterolemia and prevent cardiovascular diseaseOXFORD, ENGLAND / ACCESSWIRE / September 20, 2024 / GENinCode Plc (AIM:GENI), the genetics compa ...

LONDON, UK and CAMBRIDGE, MA, USA I September 19, 2024 I Beacon Therapeutics Holdings Limited ('Beacon' or 'the Company'), a leading ophthalmic gene therapy ...

Immunosuppressants used in adeno-associated virus (AAV) gene therapy for monogenic disorders present an adverse effect ...

and monogenic entities where senescence or alveolar stem cell exhaustion is not the primary pathogenetic mechanism. This narrative review, the first in a series of four, synthesizes current knowledge ...

Its portfolio includes promising therapies for conditions like giant axonal neuropathy, Rett syndrome, and various forms of Batten disease. Taysha Gene Therapies works closely with The University of ...

Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company focused on transforming the lives of patients living with rare neuroendocrine ...

BOSTON, Sept. 16, 2024 (GLOBE NEWSWIRE) -- (Nasdaq: RYTM), a commercial-stage biopharmaceutical company focused on ...