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 16, 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

UMN researchers give new insight to muscular dystrophy patients
(University of Minnesota Medical School) New research by University of Minnesota scientists has revealed the three dimensional structure of the DUX4 protein, which is responsible for the disease, facioscapulohumeral muscular dystrophy (FSHD). Unlike the majority of genetic diseases, FSHD is not caused by a protein that is missing or not functioning properly. Rather it is caused when a functioning, normal, protein shows up in a place where it doesn't belong (in muscles). (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - December 12, 2018 Category: International Medicine & Public Health Source Type: news

Newly published model of FSHD and a potential gene therapy to improve functional outcomes
(Nationwide Children's Hospital) Facioscapulohumeral muscular dystrophy (FSHD) is the most prevalent dominantly inherited muscular dystrophy in the world. To date, there are no pharmacologic treatments available for the more than 850,000 people affected worldwide. Developing models to use for testing potential therapeutics has been a challenge for the research community. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - November 16, 2018 Category: International Medicine & Public Health Source Type: news

FSH Society Announces Third Annual World FSHD Day to be Celebrated on...
Advocates, patient and families from around the world to unite to raise awareness for facioscapulohumeral muscular dystrophy(PRWeb June 04, 2018)Read the full story at https://www.prweb.com/releases/2018/06/prweb15528649.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - June 4, 2018 Category: Pharmaceuticals Source Type: news

University of Minnesota researchers replicate FSH muscular dystrophy in mice
(University of Minnesota) A new study published in the journal Nature Communications describes a breakthrough in research related to facioscapulohumeral muscular dystrophy (FSHD). The debilitating genetic disease -- which has no approved treatment -- affects an estimated 38,000 Americans and causes muscle degeneration. Scientists inserted into mice a gene called DUX4, which is believed to cause FSHD in humans. When they activated the gene in mice skeletal muscle cells, the animals developed a muscular dystrophy with key features of FSHD. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - September 15, 2017 Category: International Medicine & Public Health Source Type: news

aTyr Pharma Announces Appointment of Jeffery S. Hatfield to its Board of Directors
SAN DIEGO, April 3, 2017 -- (Healthcare Sales & Marketing Network) -- aTyr Pharma, Inc. (LIFE), a biotherapeutics company engaged in the discovery and development of Physiocrine-based therapeutics to address severe, rare diseases, today announced the appo... Biopharmaceuticals, Personnel aTyr Pharma, Physiocrine, Resolaris, facioscapulohumeral muscular dystrophy (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - April 3, 2017 Category: Pharmaceuticals Source Type: news

Medical News Today: Muscular dystrophy: Cancer drug may boost muscle strength for some patients
Researchers reveal how the cancer drug sunitinib may reduce muscle weakness for patients with facioscapulohumeral dystrophy - a form of muscular dystrophy. (Source: Health News from Medical News Today)
Source: Health News from Medical News Today - November 22, 2016 Category: Consumer Health News Tags: Muscular Dystrophy / ALS Source Type: news

How Controversial Gene Editing Could Lead To Groundbreaking Cures
Thanks to the controversial new technology known as CRISPR, scientists are beginning to make headway in understanding and potentially curing some of the world's most intractable diseases. Sickle-cell anemia, HIV, schizophrenia and autism -- essentially, anything involving bad DNA is now fair game. The latest example, from a study published earlier this month in the journal Molecular Therapy, focuses on Facioscapulohumeral muscular dystrophy, or FSHD, which is one of the most common forms of muscular dystrophy. The genetic disease causes the muscle fibers in the face, shoulders and upper arms to weaken over time -- and...
Source: Science - The Huffington Post - November 28, 2015 Category: Science Source Type: news

aTyr Pharma begins Resolaris Phase Ib/II trial to treat early onset FSHD
aTyr Pharma has started a Phase Ib/II trial of Resolaris for patients with early onset facioscapulohumeral muscular dystrophy (FSHD). (Source: Drug Development Technology)
Source: Drug Development Technology - November 10, 2015 Category: Pharmaceuticals Source Type: news

Gene discovery could lead to muscular dystrophy treatment
Researchers have made a critical discovery about a gene involved in muscular dystrophy that could lead to future therapies for the currently untreatable disease. Facioscapulohumeral muscular dystrophy (FSHD) is a progressive wasting disease that affects the face, arms and shoulders. It is most commonly diagnosed in teenage or early adults, and though it is rarely fatal is it very debilitating. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - June 17, 2015 Category: Science Source Type: news

aTyr Pharma’s Resolaris obtains EMA orphan drug designation to treat FSHD
Biotherapeutics firm aTyr Pharma has received orphan drug designation from the European Commission (EC) for its Resolaris to treat facioscapulohumeral muscular dystrophy (FSHD). (Source: Pharmaceutical Technology)
Source: Pharmaceutical Technology - February 19, 2015 Category: Pharmaceuticals Source Type: news

Researchers find animal model for understudied type of muscular dystrophy
An animal research model has been developed for facioscapulohumeral muscular dystrophy (FSHD) to be used for muscle regeneration research as well as studies of the effectiveness of potential therapies for FSHD. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - August 28, 2014 Category: Science Source Type: news

UMN researchers find animal model for understudied type of muscular dystrophy
(University of Minnesota Academic Health Center) Researchers at the University of Minnesota have developed an animal research model for facioscapulohumeral muscular dystrophy (FSHD) to be used for muscle regeneration research as well as studies of the effectiveness of potential therapies for FSHD.The research is published in the current edition of the journal Cell Reports. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - August 28, 2014 Category: Global & Universal Source Type: news

Successful regeneration of human skeletal muscle in mice
Researchers recently announced study findings showing the successful development of a humanized preclinical model for facioscapulohumeral muscular dystrophy, providing scientists with a much needed tool to accelerate novel therapeutic research and development. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - January 27, 2014 Category: Science Source Type: news

The FSH Society Answers Questions About the New Gene for FSH Muscular...
Fueled by funding from the FSH Society, an international research team has discovered the genes that cause facioscapulohumeral muscular dystrophy (FSHD), a devastating muscle-wasting disease that...(PRWeb January 24, 2013)Read the full story at http://www.prweb.com/releases/2013/1/prweb10348916.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - January 25, 2013 Category: Pharmaceuticals Source Type: news