EMERYVILLE, Calif., Oct. 02, 2020 (GLOBE NEWSWIRE) — Zogenix, Inc. (NASDAQ:ZGNX), a global biopharmaceutical company developing and commercializing rare disease therapies, and its subsidiary Modis Therapeutics, shared new data and information about MT1621, the company’s investigational therapy for Thymidine kinase 2 deficiency (TK2d), at this week’s International Congress of the World Muscle Society (WMS 2020). TK2d is an inherited mitochondrial DNA depletion syndrome that primarily affects infants and children and is often fatal.
“These new analyses highlight the unmet need in TK2 deficiency and underscore the important progress we are making in our MT1621 development program,” said Joanne Quan, M.D., Chief Medical Officer for Modis Therapeutics. “We are excited to be collaborating with researchers and clinicians internationally to understand and develop a treatment for this devastating disease.”
The company’s WMS 2020 posters, now available on the Zogenix Newsroom, include:
- MOA: MT1621 for Thymidine kinase 2 deficiency (TK2d) Mechanism of Action
CAMBRIDGE, Mass., Oct. 01, 2020 (GLOBE NEWSWIRE) — Fulcrum Therapeutics, Inc. (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced it will present multiple posters on the company’s ongoing studies in patients with facioscapulohumeral muscular dystrophy (FSHD) during the 25th International Congress of the World Muscle Society.
“FSHD is a serious and debilitating disease for which there are currently no approved therapies,” said Diego Cadavid, MD, Fulcrum’s senior vice president, clinical development. “We are pleased to share these data at this important scientific meeting as we continue to pursue losmapimod as a potential treatment for FSHD by addressing the root cause of the disease. We greatly appreciate the patients who have participated in our trials and the support we have received from key opinion leaders and investigators.”
During the Virtual Poster Session today, October 1, 2020 from 12:30pm
CAMBRIDGE, Mass.–(BUSINESS WIRE)–Sep 30, 2020–
Scholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced two poster presentations as part of the World Muscle Society (WMS) 2020 Virtual Congress. The SRK-015 clinical development poster will showcase previously presented data from the Phase 1 healthy volunteer trial, as well as baseline characteristics and demographics from the TOPAZ Phase 2 proof-of-concept trial evaluating SRK-015 for the treatment of patients with Type 2 and Type 3 Spinal Muscular Atrophy (SMA). The second poster shares data on the pharmacologic effects of SRK-015 in patients with SMA, healthy volunteers, and across animal species.
Details for the two virtual posters at the WMS meeting are as follows:
- Title: Clinical Development of SRK-015, a Fully Human Anti-proMyostatin Monoclonal Antibody, for the Treatment of Later-Onset Spinal Muscular Atrophy
- Title: Myostatin
STRASBOURG, Pa. and PHILADELPHIA, Sept. 28, 2020 /PRNewswire/ — Dynacure, a clinical stage drug development company focused on improving the lives of patients with rare and orphan disorders, announced today that the company will present two posters containing data on the natural history of Myotubular and Centronuclear Myopathies (CNM) at the 25th Annual Congress of the World Muscle Society (WMS25). The WMS25 meeting is being held in a virtual format.
Dynacure is developing DYN101, an investigational antisense medicine designed to modulate the expression of dynamin 2 (DNM2), for the treatment of CNM. DYN101 is currently being evaluated in a Phase 1/2 clinical study, ‘UNITE-CNM’ (DYN101-C101), at multiple clinical sites in Europe.
Details of the poster presentations are listed below:
Poster Abstract: # LSP (late submitted poster) 6
Title: Hierarchical Bayesian model of disease progression in centronuclear myopathy allows to demonstrate treatment efficacy with a small sample size