Facioscapulohumeral Muscular Dystrophy News and Research

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Toxic protein may be the cause of muscular dystrophy and arhinia

Balancing care responsibilities with paid work provides working caregivers with emotional benefits

19 Feb 2023

NUS researchers discover a new way to interpret unsolved Mendelian diseases

The development of an embryo is a well-orchestrated string of processes, ensuring correct formation and positioning of vital organs of the growing organism.

29 Jun 2022

Novel promising biomarker correlates with the severity of facioscapulohumeral muscular dystrophy

There is currently no effective treatment for facioscapulohumeral muscular dystrophy (FSHD), one of the most common neuromuscular diseases, which is caused by an abnormal expression of the transcription factor DUX4.

15 Feb 2022

New initiative to reduce treatment delays for patients with rare diseases

The Treatabolome project is a research initiative to develop a freely available, interoperable online platform dedicated to disseminating rare disease and gene-specific treatment information to healthcare professionals regardless of their level of specialized expertise.

1 Jun 2021

Mother's protein found to be involved in genomic imprinting process

Mothers leave their mark on their children in many ways - and Australian researchers have discovered a protein called SMCHD1 is involved in this 'imprinting' process.

24 Nov 2020

Researchers develop potential new therapy for common form of muscular dystrophy

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.

29 Jun 2020

SLU research halts toxic protein linked to muscular dystrophy in animal models

There is no cure or treatment for facioscapulohumeral muscular dystrophy, an illness that starts with facial, shoulder and upper arm weakness and eventually affects nearly all skeletal muscles of those with the disease.

23 Oct 2019

Study identifies inhibitor that protects cells from toxic DUX4 effects

About one in 8,000 people have facioscapulohumeral muscular dystrophy, according to a 2014 study, which is relatively common in the world of genetic diseases.

11 Sep 2019

New model of FSHD could be useful to study effectiveness of experimental therapeutics

Facioscapulohumeral muscular dystrophy 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.

17 Nov 2018

MDA announces 34 new grants totaling $9.9 million to accelerate neuromuscular research

The Muscular Dystrophy Association today announced the award of 34 new grants totaling more than $9.9 million for its summer round of funding. These new grants represent a continued commitment by MDA to fund groundbreaking research that will accelerate treatments and cures for the more than 40 diseases in its program.

24 Aug 2018

SLU researcher reports success in identifying new drug targets for muscular dystrophy

In a recent paper published in the journal Skeletal Muscle, a Saint Louis University researcher reports success in identifying new drug targets that potentially could slow or halt the progression of a form muscular dystrophy, an illness characterized by progressive muscle degeneration.

7 Nov 2017

University Of Minnesota scientists describe breakthrough in research related to FSHD

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 degeneration and wasting of the skeletal muscles.

15 Sep 2017

First patient dosed in long term-safety extension study of Resolaris for adult patients with FSHD

aTyr Pharma, Inc., a biotherapeutics company engaged in the discovery and development of Physiocrine-based therapeutics to address severe rare diseases, today announced that the first patient has been dosed in a newly initiated long term-safety extension study of Resolaris for adult patients with facioscapulohumeral muscular dystrophy (FSHD), a rare and severe genetic myopathy for which there are currently no approved treatments.

10 Sep 2015

SHIFT Communications expands healthcare practice

SHIFT Communications, the leader in data-driven public relations and communications, today announced the expansion of its healthcare practice. Since January, SHIFT's healthcare practice has grown its revenue nearly 30 percent and expanded not only the team, but the diversity of the clientele they serve – moving beyond health information technology (IT) to sectors ranging from digital health to the life sciences.

23 Jun 2015

Australian researchers discover gene involved in muscular dystrophy

Australian researchers have made a critical discovery about a gene involved in muscular dystrophy that could lead to future therapies for the currently untreatable disease.

18 Jun 2015

aTyr's Resolaris granted FDA Orphan Drug Designation for treatment of FSHD

aTyr Pharma, a biotherapeutics company engaged in the discovery and development of Physiocrine-based therapeutics to address rare diseases, announced today that Resolaris has been granted Orphan Drug Designation by the U.S. Food and Drug Administration for the treatment of facioscapulohumeral muscular dystrophy (FSHD).

27 Apr 2015

EC grants orphan drug designation to aTyr Pharma's Resolaris for treatment of FSHD

aTyr Pharma, Inc., a biotherapeutics company engaged in the discovery and development of Physiocrine-based therapeutics to address rare diseases, announced today the European Commission (EC) has granted orphan drug designation to Resolaris for the treatment of facioscapulohumeral muscular dystrophy (FSHD).

19 Feb 2015

Minnesota researchers develop animal research model for FSHD

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.

29 Aug 2014

Researchers develop valid humanized model for facioscapulohumeral muscular dystrophy

Researchers at the Kennedy Krieger Institute recently announced study findings showing the successful development of a humanized preclinical model for facioscapulohumeral muscular dystrophy (FSHD), providing scientists with a much needed tool to accelerate novel therapeutic research and development.

28 Jan 2014

DART Therapeutics develops new class of therapy for Duchenne muscular dystrophy

DART Therapeutics Inc., an innovative, new-model biotechnology firm focused on developing therapies for Duchenne muscular dystrophy, announced today that it is developing a SARM drug candidate obtained from Belgium-based Galapagos NV.

21 Mar 2013