Rare Disease Day 2024 – Call for greater awareness of X-linked adrenoleukodystrophy and cerebral adrenoleukodystrophy – special film launched

2024-02-29 Rare Disease Day 2024 – Call for greater awareness of X-linked adrenoleukodystrophy and cerebral adrenoleukodystrophy – special film launched Barcelona, Spain and Düsseldorf, Germany – 29 February, 2024 – Neuraxpharm Group (Neuraxpharm), a leading European specialty pharmaceutical company focused on the treatment of central nervous system (CNS) disorders, and Minoryx Therapeutics, a late stage biotech…

Aura Biosciences to Participate in Upcoming Investor Conferences

Aura Biosciences to Participate in Upcoming Investor ConferencesFebruary 26, 2024PDF Version BOSTON–(BUSINESS WIRE)–Feb. 26, 2024– Aura Biosciences Inc. (NASDAQ: AURA), a clinical-stage biotechnology company developing a novel class of virus-like drug conjugate (VDC) therapies for multiple oncology indications, today announced that Elisabet de los Pinos, PhD, Chief Executive Officer, will participate in the following upcoming investor conferences: 44th Annual TD…

Reneo Pharmaceuticals Announces Results from Pivotal STRIDE Study of Mavodelpar in Primary Mitochondrial Myopathies (PMM)

December 14, 2023 The STRIDE study did not meet its primary or secondary efficacy endpoint The company to suspend mavodelpar development and implement a workforce reduction IRVINE, Calif., Dec. 14, 2023 (GLOBE NEWSWIRE) — Reneo Pharmaceuticals, Inc. (NASDAQ: RPHM), a pharmaceutical company focused on the development and commercialization of therapies for patients with rare genetic…

Update on Regulatory Review of leriglitazone in the EU

2024-01-26 A re-examination for cerebral adrenoleukodystrophy (cALD) will be requested following the negative CHMP opinion on X-linked adrenoleukodystrophy (X-ALD) Barcelona, Spain and Düsseldorf, Germany – 26 January, 2024 – Minoryx Therapeutics, a late stage biotech company focused on the development of therapies for orphan central nervous system (CNS) disorders and Neuraxpharm Group (Neuraxpharm), a leading European…

Glycomine Announces First Pediatric Patient Dosed in Phase 2 Clinical Study of GLM101, a Potential Treatment for PMM2-CDG

DECEMBER 11, 2023 SAN CARLOS, Calif., December 11, 2023 – Glycomine, Inc., a clinical-stage biotechnology company focused on developing new therapies for orphan diseases, announced the first pediatric patient has been dosed in a multi-center open-label Phase 2 study of GLM101, a mannose-1-phosphate replacement therapy, in patients with phosphomannomutase 2-congenital disorder of glycosylation (PMM2-CDG). “The initiation of dosing…

Aura Biosciences Announces First Patient Dosed in Global Phase 3 CoMpass Trial Evaluating the Safety and Efficacy of Belzupacap Sarotalocan (Bel-sar) for First-Line Treatment of Early-Stage Choroidal Melanoma

December 7, 2023 BOSTON–(BUSINESS WIRE)–Dec. 7, 2023– Aura Biosciences Inc. (NASDAQ: AURA), a clinical-stage biotechnology company developing a novel class of virus-like drug conjugate (VDC) therapies for multiple oncology indications, today announced the first patient has been dosed in the global Phase 3 CoMpass trial evaluating the safety and efficacy of bel-sar for the first-line treatment of…

Reneo Pharmaceuticals to Participate in Upcoming Investor Conferences

November 2, 2023 IRVINE, Calif., Nov. 02, 2023 (GLOBE NEWSWIRE) — Reneo Pharmaceuticals, Inc. (NASDAQ: RPHM), a clinical stage pharmaceutical company focused on the development and commercialization of therapies for patients with rare genetic mitochondrial diseases, today announced that the company’s management team will participate in the following investor conferences. Jefferies London Healthcare ConferenceDate: Tuesday,…

Reneo Pharmaceuticals Reports Third Quarter 2023 Financial Results

November 13, 2023 Topline data results from the pivotal STRIDE study are expected in December 2023 IRVINE, Calif., Nov. 13, 2023 (GLOBE NEWSWIRE) — Reneo Pharmaceuticals, Inc. (Nasdaq: RPHM), a clinical-stage pharmaceutical company focused on the development and commercialization of therapies for patients with rare genetic mitochondrial diseases, today reported financial results for the third…

Minoryx announces enrollment of first patients with cerebral Adrenoleukodystrophy (cALD) in US Phase 3 clinical trial, CALYX

2023-11-16 As part of CALYX, Minoryx is conducting an extensive pre-screening MRI-based campaign aimed at identifying eligible adult X-ALD patients with cerebral Adrenoleukodystrophy (cALD) Mataró, Barcelona, Spain, November 16, 2023 – Minoryx Therapeutics, a registration stage biotech company focused on the development of therapies for orphan central nervous system (CNS) disorders, today announces the first patients have…

Glycomine Presents at ASHG 2023 Data from Ongoing Natural History Study with Insight into Genetic Mutations and Biomarkers for PMM2-CDG

NOVEMBER 2, 2023 SAN CARLOS, Calif., November 3, 2023 – Glycomine, Inc., a biotechnology company focused on developing new therapies for orphan diseases, announced the presentation of summary findings from its ongoing natural history study at the American Society for Human Genetics (ASHG) 2023 Annual Meeting taking place in Washington, DC.  The findings included genotype characterization, combined with…