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So far Patrick has created 24 blog entries.

Santhera Announces Completion of ReveraGen’s Long-Term Extension Study with Vamorolone in Duchenne Muscular Dystrophy

Santhera Announces Completion of ReveraGen’s Long-Term Extension Study with Vamorolone in Duchenne Muscular Dystrophy Announcement attached,  Please click on link to view:  2020-06-02_Vamorolone_LTE_e_final

Catabasis Pharmaceuticals present important information updates on Edasalonexent during the Muscular Dystrophy Association Virtual Poster Session

13 May 2020 Catabasis Pharmaceuticals has presented important information updates on the Edasalonexent program in Phase 3 development for the treatment of Duchenne muscular dystrophy (DMD) at the 2020 Muscular Dystrophy Association (MDA) Virtual Poster Session. The information updates presented outline the potential for Edasalonexent to provide positive effects for DMD including: Capsule swallowing abilities Expected patient population Growth

Update IGNITE DMD

Yesterday, Solid Biosciences issued a press release following receipt of written communication from the USA FDA regarding the clinical hold placed on their IGNITE DMD Phase I/II clinical trial. Please find below links to their press release and the corresponding letter they drafted to the Duchenne community. Community Letter: https://www.solidbio.com/about/media/news/letter-to-the-duchenne-community-update-on-sgt-001-phase-i-ii-clinical-hold-for-ignite-dmd-program Press Release:  https://investors.solidbio.com/news-releases/news-release-details/solid-biosciences-provides-update-regarding-sgt-001-phase-iii

The care of patients with Duchenne, Becker and other muscular dystrophies in the COVID‐19 pandemic

24 April 2020 In this paper, the authors discuss their consensus recommendations pertaining to the care of muscular dystrophy patients during the COVID-19 pandemic, in particular: Corticosteroid and exon skipping treatments Cardiac medications Hydroxychloroquine use Emergency and respiratory care Rehabilitation management Conduct of clinical trials   The importance of collaborative treatment decisions between the patient,

Catabasis Pharmaceuticals to Present at 2020 Muscular Dystrophy Association Virtual Poster Session

17 April 2020 Catabasis Pharmaceuticals will present information on the Edasalonexent program in Phase 3 development for the treatment of Duchenne muscular dystrophy (DMD) at the 2020 Muscular Dystrophy Association (MDA) Virtual Poster Session later this month. Edasalonexent is being developed as a potential foundational therapy aimed at improving the lives of all patients affected

The Importance of Involving Patients in Genomic Research

10 April 2020 Kat Arney, a biologist and award-winning science communicator, finds out why it is so important to ensure both academic and commercial research studies are done with rather than on participants, in the latest podcast of Genetics Unzipped. Podcast panel members include Fiona Copeland (chair of a support group for UK families affected

Management and Operational Plan for COVID-19 People with Disability

21 April 2020 The Management and Operational plan for COVID-19 for People with Disability developed by the Australian Commonwealth Government provides a targeted response for people with disability, their families, carers and support workers. The plan provides high-level guidance on a range of factors that need to be considered in managing and preventing the transmission

Solid Biosciences update on IGNITE DMD

This morning Solid Biosciences provided an update to the market which included new patient data from their IGNITE DMD clinical program. Please find below the press release and corresponding letter they drafted to the Duchenne community. Community Letter: https://www.solidbio.com/about/media/news/letter-to-the-duchenne-community-data-and-ignite-dmd-clinical-trial-update-1 Press Release: https://www.solidbio.com/about/media/press-releases/solid-biosciences-reports-fourth-quarter-and-full-year-2019-financial-results-and-provides-business-update

Coronavirus (COVID-19) Advice for the Duchenne & Becker Community

Save Our Sons first obligation is to our community, who are at the heart of everything we do. We are acutely aware of how important it is for people to follow the advice of experts in the health and medical science fields, the clinicians and doctors who care for our families with such passion and

Australian Neuromuscular Disease Registry

The Australian Neuromuscular Disease Registry (ANMDR) is an Australia-wide registry of people diagnosed with a neuromuscular disease. It collects important medical information from adult and child patients across the country to improve the understanding of neuromuscular disease and accelerate the development of new therapies. Save Our Sons Duchenne Foundation have played a critical role in