Facioscapulohumeral Muscular Dystrophy (FSHD) News 
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This is an RSS file. You can use it to subscribe to this data in your favourite RSS reader or to display this data on your own website or blog.[Ad hoc announcement pursuant to Art. 53 LR] Roche announces EMBARK trial in Duchenne muscular dystrophy (DMD) did not reach primary endpoint, but shows positive efficacy outcomes on all timed functional key endpoints
This study is ongoing.Study 301 (EMBARK), a Phase 3 global, randomised, double-blinded and placebo-controlled study of Elevidys in ambulatory Duchenne patients aged 4-<8 years old.The ENVOL trial (Study 302) a Phase 2 study in children with Duchenne. The study aims to enrol 21 participants who are under 4 years of age, including newborns. Not yet started.The ENVISION trial (Study 303), a Phase 3 study in older ambulatory/non-ambulatory patients which is now recruiting.The EXPEDITION long-term (5 year) follow up study (Study 305) of participants who have received Elevidys in a previous clinical study, which is not yet re...
Source: Roche Media News - October 31, 2023 Category: Pharmaceuticals Source Type: news
[Ad hoc announcement pursuant to Art. 53 LR] Roche announces EMBARK trial in Duchenne muscular dystrophy (DMD) did not reach primary endpoint, but shows positive efficacy outcomes on all timed functional key endpoints
This study is ongoing.Study 301 (EMBARK), a Phase 3 global, randomised, double-blinded and placebo-controlled study of Elevidys in ambulatory Duchenne patients aged 4-<8 years old.The ENVOL trial (Study 302) a Phase 2 study in children with Duchenne. The study aims to enrol 21 participants who are under 4 years of age, including newborns. Not yet started.The ENVISION trial (Study 303), a Phase 3 study in older ambulatory/non-ambulatory patients which is now recruiting.The EXPEDITION long-term (5 year) follow up study (Study 305) of participants who have received Elevidys in a previous clinical study, which is not yet re...
Source: Roche Investor Update - October 31, 2023 Category: Pharmaceuticals Source Type: news
The FSHD Society Celebrates Fulcrum's Milestone in the Fight Against FSHD
Fulcrum Therapeutics has announced successful completion of patient enrollment in REACH Phase 3 trial RANDOLPH, Mass., Sept. 8, 2023 /PRNewswire-PRWeb/ -- The FSHD Society, a leading advocate and partner in the quest to find treatments for Facioscapulohumeral Muscular Dystrophy (FSHD), is... (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - September 8, 2023 Category: Pharmaceuticals Tags: TRI Source Type: news
World FSHD Alliance annual summit convenes in Milan, Italy
Leaders from patient advocacy groups from around the world meet to advance clinical trial readiness and access to treatments for facioscapulohumeral muscular dystrophy(PRWeb June 19, 2023)Read the full story at https://www.prweb.com/releases/world_fshd_alliance_annual_summit_convenes_in_milan_italy/prweb19386094.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - June 19, 2023 Category: Pharmaceuticals Source Type: news
FSHD1 Rare Neuromuscular Disorder Diagnosis Brings Answers for Teen
A rare neuromuscular disorder diagnosis of Facioscapulohumeral Muscular Dystrophy (FSHD) brings long awaited answers for teenager (Source: Disabled World)
Source: Disabled World - May 1, 2023 Category: Disability Tags: Muscular Dystrophy (MD) Source Type: news
Roche to present new data from its expanding neuromuscular disease portfolio at World Muscle Society 2022
New positive data fromEvrysdi, a treatment for spinal muscular atrophy (SMA), a progressive neuromuscular disease that can be fatalData from the gene therapyprogramme forDuchenne muscular dystrophy (DMD), a progressive disease that leads to premature death, reinforce confidence in the most advanced Phase 3 study currently underwayStudy designs of two new trials ingeneralised myasthenia gravis (gMG), a rare chronic autoimmune disease, andfacioscapulohumeral muscular dystrophy (FSHD), a genetic muscle disorderBasel, 05 October 2022 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that new data from its industry-leading n...
Source: Roche Media News - October 5, 2022 Category: Pharmaceuticals Source Type: news
Roche to present new data from its expanding neuromuscular disease portfolio at World Muscle Society 2022
New positive data fromEvrysdi, a treatment for spinal muscular atrophy (SMA), a progressive neuromuscular disease that can be fatalData from the gene therapyprogramme forDuchenne muscular dystrophy (DMD), a progressive disease that leads to premature death, reinforce confidence in the most advanced Phase 3 study currently underwayStudy designs of two new trials ingeneralised myasthenia gravis (gMG), a rare chronic autoimmune disease, andfacioscapulohumeral muscular dystrophy (FSHD), a genetic muscle disorderBasel, 05 October 2022 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that new data from its industry-leading n...
Source: Roche Investor Update - October 5, 2022 Category: Pharmaceuticals Source Type: news
AMRA joins clinical trial on muscular dystrophy
AMRA Medical will be involved in the Facioscapulohumeral Muscular Dystroph...Read more on AuntMinnie.comRelated Reading:
AMRA touts clinical trial results
AMRA adds UK Biobank images to database
AMRA teams up with National University of Singapore
AMRA highlights body composition analysis research
AMRA wins FDA clearance for MR software (Source: AuntMinnie.com Headlines)
Source: AuntMinnie.com Headlines - October 15, 2021 Category: Radiology Source Type: news
Mother and son share rare muscle disorder, treated decades apart by same doctor
Twenty-four years ago, Bobbi Heinold, 17, was diagnosed with facioscapulohumeral muscular dystrophy, a genetic muscle condition characterized by weakness, especially in the face, shoulder blades and upper arms. Doctors told her it could affect her quality of life. Bobbi and her mom drove from their Indiana home to Mayo Clinic in Rochester, Minnesota, to learn [...] (Source: News from Mayo Clinic)
Source: News from Mayo Clinic - May 6, 2021 Category: Databases & Libraries Source Type: news
New treatment for common form of muscular dystrophy shows promise in cells, animals
(University of Alberta Faculty of Medicine& Dentistry) Researchers have designed a potential new treatment for one of the most common forms of muscular dystrophy, according to a new study published today in the Proceedings of the National Academy of Sciences. Toshifumi Yokota, professor of medical genetics at the University of Alberta, led a team from Canada and the U.S. to create and test synthetic DNA-like molecules that interfere with the production of a toxic protein that destroys the muscles of people who have facioscapulohumeral muscular dystrophy (FSHD). (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - June 29, 2020 Category: International Medicine & Public Health Source Type: news
Facioscapulohumeral muscular dystrophy community speaks to the FDA
FSHD Society holds externally led Patient-Focused Drug Development Meeting on FSH muscular dystrophy(PRWeb June 29, 2020)Read the full story at https://www.prweb.com/releases/facioscapulohumeral_muscular_dystrophy_community_speaks_to_the_fda/prweb17220588.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - June 29, 2020 Category: Pharmaceuticals Source Type: news
Crystal structure of the hinge domain of Smchd1 reveals its dimerization mode and nucleic acid-binding residues
Structural maintenance of chromosomes flexible hinge domain containing 1 (SMCHD1) is an epigenetic regulator in which polymorphisms cause the human developmental disorder, Bosma arhinia micropthalmia syndrome, and the degenerative disease, facioscapulohumeral muscular dystrophy. SMCHD1 is considered a noncanonical SMC family member because its hinge domain is C-terminal, because it homodimerizes rather than heterodimerizes, and because SMCHD1 contains a GHKL-type, rather than an ABC-type ATPase domain at its N terminus. The hinge domain has been previously implicated in chromatin association; however, the underlying mechan...
Source: Signal Transduction Knowledge Environment - June 15, 2020 Category: Science Authors: Chen, K., Birkinshaw, R. W., Gurzau, A. D., Wanigasuriya, I., Wang, R., Iminitoff, M., Sandow, J. J., Young, S. N., Hennessy, P. J., Willson, T. A., Heckmann, D. A., Webb, A. I., Blewitt, M. E., Czabotar, P. E., Murphy, J. M. Tags: STKE Research Articles Source Type: news
FSHD Society to Hold Landmark " Voice of the Patient " Meeting
Externally led Patient-Focused Drug Development meeting on facioscapulohumeral muscular dystrophy patients will take place in April of 2020(PRWeb October 23, 2019)Read the full story at https://www.prweb.com/releases/fshd_society_to_hold_landmark_voice_of_the_patient_meeting/prweb16664359.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - October 23, 2019 Category: Pharmaceuticals Source Type: news
FSHD Society Announces 2020 International Research Congress and...
The nation’s capital will host the world’s premier conferences on facioscapulohumeral muscular dystrophy for all stakeholders(PRWeb September 10, 2019)Read the full story at https://www.prweb.com/releases/fshd_society_announces_2020_international_research_congress_and_connect_conferences/prweb16555337.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - September 10, 2019 Category: Pharmaceuticals Source Type: news
DDT COA #000090: Facioscapulohumeral Composite Functional Outcome Measure (FSHD-COM)
(Source: FDA Center for Drug Evaluation and Research - What's New)
Source: FDA Center for Drug Evaluation and Research - What's New - June 26, 2019 Category: Drugs & Pharmacology Authors: FDA Source Type: news
