Zevra Therapeutics announced the presentation of four posters highlighting positive new data on Miplyffa (arimoclomol) for the treatment of Niemann-Pick Disease Type C (NPC) at the 22nd Annual… read more.
Health Canada has issued a Notice of Compliance with conditions (NOC/c) for Voxzogo (vosoritide for injection) a modified recombinant human C-type natriuretic peptide 39 (CNP-39) for the treatment… read more.
Camurus announced that the FDA has accepted for review the company’s resubmission of the New Drug Application (NDA) for Oclaiz (CAM 2029), octreotide extended-release injection, for the treatment… read more.
Arrowhead Pharmaceuticals, Inc. announced that the Chinese National Medical Products Administration (NMPA) has approved Redemplo (plozasiran) for the reduction of triglyceride levels in adult patients with familial chylomicronemia… read more.
Abeona Therapeutics Inc. announced the first patient treatment with FDA-approved Zevaskyn (prademagene zamikeracel), a first-of-its-kind, autologous gene therapy for treating wounds in adult and pediatric patients with recessive… read more.
A test using genetics to predict who is at the highest risk of developing type 1 diabetes could one day be applied to largescale health studies, to identify… read more.
The UK Centre of Excellence in Regulatory Science and Innovation in Pharmacogenomics (UK CERSI-PGx), led by the University of Liverpool, has published its first clinical guideline focused on… read more.
Arrowhead Pharmaceuticals, Inc. announced that the FDA has approved Redemplo (plozasiran), a small interfering RNA (siRNA) medicine, as an adjunct to diet to reduce triglycerides in adults with… read more.
Regeneron Pharmaceuticals, Inc. announced that the FDA has approved Evkeeza (evinacumab-dgnb) ANGPTL3 antibody as an adjunct to diet and exercise and other lipid-lowering therapies for the treatment of… read more.
NICE (UK) – Garadacimab can be used as an option to prevent recurrent attacks of hereditary angioedema in people 12 years and over, only if: i) they have… read more.
Mayo Clinic researchers have developed a genetic test that can help predict how people will respond to weight loss medications such as GLP-1s. The test estimates an individual’s… read more.
In a Policy Forum, Natalie Ram and colleagues discuss the concerning gaps in robust regulatory protection on direct-to-consumer genetic data and biospecimens. After declaring bankruptcy in March 2025,… read more.
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