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muscle disease

This page shows the latest muscle disease news and features for those working in and with pharma, biotech and healthcare.

Orphan drugs from Biogen and Amicus win UK Prix Galien

Orphan drugs from Biogen and Amicus win UK Prix Galien

Galafold (migalastat) is an oral medication for the treatment of Fabry disease, a rare lysosomal metabolic storage condition. ... a cruel muscle-wasting rare disease in children.

Latest news

  • Duchenne UK backs Evox dystrophin delivery project Duchenne UK backs Evox dystrophin delivery project

    Duchenne UK wants Evox to see if it can deliver full or partial lengths of dystrophin, the protein lacking in patients with the muscle-wasting disease, using its exosome technology. ... This work will also allow us to explore targeted delivery of

  • Pfizer abandons antibody for Duchenne Pfizer abandons antibody for Duchenne

    The big pharma company halted two trials of the anti-myostatin antibody, which is designed to build muscle mass and strength. ... Novartis and Morphosys gave up on their bimagrumab for muscle-wasting disease sporadic inclusion body myositis two years ago,

  • Daily Brief: Summit's $12m lifeline, Biohaven's speech anxiety pill, Crescendo's Humabody progress Daily Brief: Summit's $12m lifeline, Biohaven's speech anxiety pill, Crescendo's Humabody progress

    BHV-0223 is a sublingual formulation of riluzole, a drug already used against the muscle degenerative disease amyotrophic lateral sclerosis. ... These include troriluzole (BHV-4157), currently in late-stage clinical trials in spinocerebellar ataxia (SCA),

  • Summit plunges after Duchenne disappointment Summit plunges after Duchenne disappointment

    However while this clinical trial failure was once typical of the DMD field, there are signs of a breakthrough against the life-shortening muscle wasting disease.

  • Sarepta soars on positive Duchenne data Sarepta soars on positive Duchenne data

    the muscle wasting disease. ... DMD – a rare muscle-wasting disease that mostly affects boys and typically is fatal by age 30 – is caused by a defect in the gene coding for dystrophin, a protein found

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Latest Intelligence

  • Deal Watch February 2016 Deal Watch February 2016

    The company is developing oral biologics that deliver disease specific amino acid compositions to restore balance in disease pathways in indications such as muscle, neurodegenerative and liver diseases. ... licence. 105. †† Genexine/ Shanghai Chemo

  • Astellas: Ten years young Astellas: Ten years young

    The two new areas the company has publicly said it will be focusing on are ophthalmology and muscle disease, along with the further development of some internal compounds in ophthalmology. ... option, while in muscle disease Astellas is working with

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