https://www.bioworld.com/rss en-us http://www.bioworld.com/articles/729717 Tue, 17 Mar 2026 11:00:00 -0400 https://www.bioworld.com/articles/729717-otsukas-repinatrabit-shows-phase-ii-efficacy-in-pku-adolescents Molecule illustration http://www.bioworld.com/articles/729496 Fri, 13 Mar 2026 12:00:00 -0400 https://www.bioworld.com/articles/729496-otsukas-repinatrabit-shows-phase-ii-efficacy-in-pku-adolescents Molecule illustration http://www.bioworld.com/articles/722762 Wed, 30 Jul 2025 09:00:00 -0400 https://www.bioworld.com/articles/722762-prime-editing-could-cure-a-rare-childhood-hemiplegia-disorder Illustration of gene editing in neurons Illustration of gene editing in neurons. Credit: The Jackson Laboratory http://www.bioworld.com/articles/722613 Tue, 29 Jul 2025 09:00:00 -0400 https://www.bioworld.com/articles/722613-prime-editing-could-cure-a-rare-childhood-hemiplegia-disorder Illustration of gene editing in neurons Illustration of gene editing in neurons. Credit: The Jackson Laboratory http://www.bioworld.com/articles/720535 Fri, 23 May 2025 09:00:00 -0400 https://www.bioworld.com/articles/720535-from-mice-to-big-animal-models-in-gene-therapy-for-rare-diseases Art concept for mouse model http://www.bioworld.com/articles/716606 Wed, 22 Jan 2025 09:00:00 -0500 https://www.bioworld.com/articles/716606-an-antidote-to-cobra-venom-designed-with-ai Illustration of cobra in front of protein structures A poisonous red spitting cobra (Naja pallida) in front of a model of a toxic snake protein (orange) and a designed antitoxin protein. Credit: Kate Zvorykina/Ella Maru Studio. http://www.bioworld.com/articles/716517 Tue, 21 Jan 2025 09:00:00 -0500 https://www.bioworld.com/articles/716517-an-antidote-to-cobra-venom-designed-with-ai Illustration of cobra in front of protein structures A poisonous red spitting cobra (Naja pallida) in front of a model of a toxic snake protein (orange) and a designed antitoxin protein. Credit: Kate Zvorykina/Ella Maru Studio. http://www.bioworld.com/articles/713881 Mon, 28 Oct 2024 09:00:00 -0400 https://www.bioworld.com/articles/713881-genetic-approaches-presented-at-esgct-provide-hope-for-genodermatoses DNA double helix illustration with section being removed in red http://www.bioworld.com/articles/709436 Thu, 13 Jun 2024 09:00:00 -0400 https://www.bioworld.com/articles/709436-novel-human-medopathy-caused-by-biallelic-med16-variants Brain as light bulb filament http://www.bioworld.com/articles/695291 Mon, 20 Mar 2023 12:00:00 -0400 https://www.bioworld.com/articles/695291-rare-diseases-yield-genomic-secrets-through-new-computational-approach Awareness ribbon with zebra pattern Sometimes, hoofbeats are zebras after all. Credit: NIAID http://www.bioworld.com/articles/695222 Fri, 17 Mar 2023 12:00:00 -0400 https://www.bioworld.com/articles/695222-rare-diseases-yield-genomic-secrets-through-new-computational-approach Awareness ribbon with zebra pattern Sometimes, hoofbeats are zebras after all. Credit: NIAID Proceedings of the National Academy of Sciences on Jan. 23, 2023, a team of scientists from Frederick National Laboratory for Cancer Research and the University of California, San Francisco (UCSF) described how neurofibromin 1 (NF1) missense mutations act in a dominant negative manner through dimerization with wild-type neurofibromin.]]> http://www.bioworld.com/articles/693645 Thu, 26 Jan 2023 09:00:00 -0500 https://www.bioworld.com/articles/693645-normal-mutant-neurofibromin-variants-team-up-to-drive-severe-neurofibromatosis http://www.bioworld.com/articles/693598 Wed, 25 Jan 2023 09:00:00 -0500 https://www.bioworld.com/articles/693598-discovering-new-therapeutic-targets-for-marfan-syndrome Various pills laid across a target http://www.bioworld.com/articles/693552 Mon, 23 Jan 2023 09:00:00 -0500 https://www.bioworld.com/articles/693552-epigenomic-editing-reactivates-neurons-in-rett-syndrome Pediatric brain illustration http://www.bioworld.com/articles/689634 Wed, 14 Sep 2022 09:00:00 -0400 https://www.bioworld.com/articles/689634-nacuity-and-arctic-therapeutics-partner-on-npi-001-for-hereditary-cystatin-c-amyloid-angiopathy http://www.bioworld.com/articles/516803 Wed, 09 Mar 2022 13:00:00 -0500 https://www.bioworld.com/articles/516803-brightinsight-and-ucb-tackle-rare-diseases-with-digital-solutions Concept of business partnership http://www.bioworld.com/articles/515705 Wed, 02 Feb 2022 12:00:00 -0500 https://www.bioworld.com/articles/515705-therapy-for-ultra-rare-xlhed-edges-closer-as-pivotal-trial-begins http://www.bioworld.com/articles/515425 Tue, 25 Jan 2022 12:00:00 -0500 https://www.bioworld.com/articles/515425-nice-set-to-expand-scope-of-htas-but-industry-still-sees-gaps U.K. flag on stethoscope http://www.bioworld.com/articles/515396 Mon, 24 Jan 2022 13:00:00 -0500 https://www.bioworld.com/articles/515396-nice-set-to-expand-scope-of-htas-but-industry-still-sees-gaps U.K. flag on stethoscope http://www.bioworld.com/articles/513247 Wed, 10 Nov 2021 12:00:00 -0500 https://www.bioworld.com/articles/513247-uk-study-illuminates-value-of-whole-genome-sequencing-in-rare-disease-care DNA NGS genome sequencing http://www.bioworld.com/articles/512172 Thu, 07 Oct 2021 13:00:00 -0400 https://www.bioworld.com/articles/512172-centogene-and-twist-bioscience-team-up-to-develop-rare-disease-tests Deal illustration http://www.bioworld.com/articles/511890 Wed, 29 Sep 2021 12:00:00 -0400 https://www.bioworld.com/articles/511890-astrazeneca-shares-soar-after-500m-swoop-for-rare-disease-firm-caelum Stock chart, upward arrow http://www.bioworld.com/articles/510761 Tue, 24 Aug 2021 12:00:00 -0400 https://www.bioworld.com/articles/510761-a-3b-deal-with-roche-shapes-up-as-one-of-the-years-biggest Handshake with DNA, molecules http://www.bioworld.com/articles/509541 Tue, 20 Jul 2021 17:00:00 -0400 https://www.bioworld.com/articles/509541-first-therapy-for-rare-liver-disease-pfic-as-albireos-bylvay-earns-priority-fda-nod http://www.bioworld.com/articles/509227 Mon, 12 Jul 2021 01:00:00 -0400 https://www.bioworld.com/articles/509227-prothena-sells-attr-program-to-novo-in-deal-worth-up-to-12b Handshake http://www.bioworld.com/articles/509187 Fri, 09 Jul 2021 12:00:00 -0400 https://www.bioworld.com/articles/509187-nice-rejects-orchards-rare-disease-drug-but-zolgensma-u-turn-offers-hope http://www.bioworld.com/articles/508127 Fri, 11 Jun 2021 12:00:00 -0400 https://www.bioworld.com/articles/508127-vertex-goes-back-to-drawing-board-after-axing-another-phase-ii-drug Liver and lungs http://www.bioworld.com/articles/507862 Fri, 04 Jun 2021 16:45:00 -0400 https://www.bioworld.com/articles/507862-liminals-rare-disease-treatment-ryplazim-is-approved-by-fda http://www.bioworld.com/articles/504169 Mon, 01 Mar 2021 12:30:00 -0500 https://www.bioworld.com/articles/504169-bridgebios-origin-secures-fda-approval-for-mocd-type-a-therapy http://www.bioworld.com/articles/503763 Thu, 18 Feb 2021 12:05:00 -0500 https://www.bioworld.com/articles/503763-evoxs-exosome-platform-draws-new-investors-in-954m-series-c Hand holding gear, dollar sign