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Articles by Solid Biosciences
Outcome monitoring and intermediate endpoints for gene therapy
Get full text access Log in, subscribe or purchase for full access. REFERENCES 1. Jessup, M. ∙ Greenberg, B. ∙ Mancini, D. ... Calcium Upregulation by Percutaneous Administration of Gene Therapy in Cardiac Disease (CUPID): a phase 2 trial of intracoronary gene therapy of sarcoplasmic reticulum Ca2+-ATPase in patients with advanced heart failure Circulation. 2011 Jul; 124:304-313 2. Greenberg, B. ∙ Butler, J. ∙ Felker, G.M. ...
11.21.23 -- Respiratory Viral Infections: The Next Frontier For Antiviral Drug Development
There is still no approved antiviral therapy for adults with RSV infections. As drug developers, we need to prioritize developing new antiviral therapies for immunocompromised patients. Complex Molecules, Complex Problems The support of an experienced CDMO is critical to overcoming complexity when working with a highly potent active pharmaceutical ingredient (HPAPI) and when solving solubility challenges.
Prolonged voluntary wheel running reveals unique adaptations in mdx mice treated with microdystrophin constructs ± the nNOS-binding site
1Department of Human Nutrition, Foods and Exercise and Metabolism Core, Virginia Tech, Blacksburg, VA, United States 2Department of Pathology and Neuroscience Research Center, Medical College of Wisconsin and Diverge Translational Science Laboratory, Milwaukee, WI, United States 3Solid Biosciences, Inc., Cambridge, MA, United States We tested the effects of prolonged voluntary wheel running on the muscle function of mdx mice treated with one of two different microdystrophin constructs.
06.13.23 -- How Wearables Enabled A Novel Endpoint For Duchenne Trials
You're receiving the Tuesday edition of the Clinical Leader newsletter, focusing on Clinical Trial Technology. To make changes to your newsletter selections, update your topic preferences. Clinical Sites | Patient Centricity | Patient Recruitment (Monday) Clinical Trial Technology (Tuesday) Decentralized Trials | Trial Management (Wednesday) Outsourcing Models | Regulatory & Compliance (Thursday) (Friday) Learn more about our personalized newsletters here.
06.07.23 -- Cell & Gene Best Of May
Hello readers, I’m happy to bring you May’s most-clicked articles. Enjoy! Erin Harris, editor in chief
05.17.23 -- FDA Inspections In India For Tablet Press Operations
By Fred A. Rowley Over the past few years, the FDA investigators in India have shifted their focus away from laboratory and data manipulation and toward a closer look at tablet press operations. The new interest centers on critical process parameters, the automatic weight control system, and more.
05.17.23 -- FDA's Project Orbis Gathers Momentum For Cell & Gene Therapies
By Jessie Hanrahan, Solid Biosciences Project Orbis has worked very well for oncology products. CBER’s director, Dr. Peter Marks, has recently started promoting the development of a similar program, which would coordinate review with other regulators, for cell and gene therapies. This would be an enormous step forward.
05.12.23 -- Project Orbis For Cell & Gene Therapies Gathers Momentum
By Jessie Hanrahan, Solid Biosciences Project Orbis has worked very well for oncology products. CBER Director Dr. Peter Marks recently started promoting the development of a similar program, which would coordinate review with other regulators, for cell and gene therapies. This would be an enormous step forward.
05.02.23 -- Project Orbis For CGTs Gathers Momentum
By Sanjay Srivastava, Global Cell and Gene Therapy Lead; Stas Verberkt, North America Life Sciences Security Lead; Lena Walton, Cell and Gene Therapy Consultant; Zach Grieco, Life Sciences Security Senior Manager; and Mike Van Chau, OT Security Senior Manager at Accenture Depending on the size of the organization, the transparency of its CGT R&D laboratories, and the timeframe for accelerated approval, the concept of building cyber resiliency in these spaces may seem daunting.
Project Orbis For Cell & Gene Therapies Gathers Momentum Original
Get more Cell & Gene insight with our FREE newsletter sign me up Discovery Trials Regulatory Outsourcing Commercialization Guest Column | April 25, 2023 By Jessie Hanrahan, Solid Biosciences Last year I was invited to moderate a panel at the Alliance for Regenerative Medicine’s Cell and Gene Meeting on the Mesa and though much was discussed, one idea seemed to catch fire: applying the FDA Oncology Center of Excellence’s Project Orbis to cell and gene therapies (CGT) regulated within CBER.
03.23.22 -- Defining Meaningful Outcomes In Gene Therapy Trials For Duchenne Muscular Dystrophy
Article | By Jo Zhang and Fiona Yeung, TransPerfect Digital health has been a trending topic in the life sciences space and has gained particular momentum due to the COVID-19 outbreak. Discover how China has remained the leader in the digital health revolution.
03.08.22 -- Duchenne Muscular Dystrophy: Defining Outcomes In GT Trials
Newsletter | March 8, 2022 By Carl A. Morris, Roxana Donisa Dreghici, and J. Patrick Gonzalez, Solid Biosciences This article discusses the limitations of classical therapeutic endpoints and how to consider novel strategies for Duchenne muscular dystrophy (DMD). By Anthony J. Coyle, Ph.D., Repertoire Immune Medicines The potential of T-cell therapies to be curative by harnessing our own immune repertoire has exciting implications for cancer and many other immune-mediated disorders.
Defining Meaningful Outcomes In Gene Therapy Trials For Duchenne Muscular Dystrophy
By Carl A. Morris, Roxana Donisa Dreghici, and J. Patrick Gonzalez, Solid Biosciences Duchenne muscular dystrophy (DMD), the most common form of muscular dystrophy, is an X-linked recessive disease that affects approximately one in 5,000 live male births and is diagnosed in about 20,000 children globally each year.1 The disease results from mutations in the DMD gene that lead to the absence of functional dystrophin protein across skeletal and cardiac muscles.
Defining Meaningful Outcomes In Gene Therapy Trials For Duchenne Muscular Dystrophy
By Carl A. Morris, Roxana Donisa Dreghici, and J. Patrick Gonzalez, Solid Biosciences Duchenne muscular dystrophy (DMD), the most common form of muscular dystrophy, is an X-linked recessive disease that affects approximately one in 5,000 live male births and is diagnosed in about 20,000 children globally each year.1 The disease results from mutations in the DMD gene that lead to the absence of functional dystrophin protein across skeletal and cardiac muscles.
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...HIGH WIND WARNING IN EFFECT FROM 3 PM THIS AFTERNOON TO 11 PM EST THIS EVENING... * WHAT...West winds 25 to 35 mph with gusts up to 60 mph expected. * WHERE...Cattaraugus and Allegany counties. * WHEN...From 3 PM this afternoon to 11 PM EST this evening. * IMPACTS...Damaging winds will blow down trees and power lines. Scattered power outages are expected. Some property damage possible. Travel will be difficult, especially for high profile vehicles. PRECAUTIONARY/PREPAREDNESS ACTIONS...
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NEW YORK, Dec. 7, 2021 /PRNewswire/ -- InvestorsObserver issues critical PriceWatch Alerts for GEVO, CCL, ARDX, SRC, and SLDB. To see how InvestorsObserver's proprietary scoring system rates these stocks, view the InvestorsObserver's PriceWatch Alert by selecting the corresponding link. (Note: You may have to copy this link into your browser then press the [ENTER] key.)
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So often, when a film starts off stating that it's "Based on" or "Inspired by" a true story, I find that I'm bracing myself, preparing to jot down notes on certain overly dramatic incidents, then Google them when I get home, proving that the film took too many liberties with the facts. I've always known that it's a pointless exercise. If I want the absolute truth in a movie, I'll stick with documentaries (though many of those are suspect, too).
Thinking about buying stock in Solid Biosciences, Enlivex Therapeutics, Sirius XM, Sundial Growers, or E*TRADE Financial?
TownNews.com Content Exchange NEW YORK, Oct. 1, 2020 /PRNewswire/ -- InvestorsObserver issues critical PriceWatch Alerts for SLDB, ENLV, SIRI, SNDL, and ETFC. To see how InvestorsObserver's proprietary scoring system rates these stocks, view the InvestorsObserver's PriceWatch Alert by selecting the corresponding link.
The Glycoprotein of the Live-Attenuated Junin Virus Vaccine Strain Induces Endoplasmic Reticulum Stress and Forms Aggregates prior to Degradation in the Lysosome
Vaccines and Antiviral Agents John T. Manning, Nadya E. Yun, Alexey V. Seregin, Takaaki Koma, Rachel A. Sattler, Chiomah Ezeomah, Cheng Huang, Juan C. de la Torre, Slobodan Paessler Mark T. Heise, Editor DOI: 10.1128/JVI.01693-19 ABSTRACT Argentine hemorrhagic fever is a potentially lethal disease that is caused by Junin virus (JUNV). There are currently around 5 million individuals at risk of infection within regions of endemicity in Argentina.
Respiratory and upper limb function as outcome measures in ambulant and non-ambulant subjects with Duchenne muscular dystrophy: a prospective multicentre study
To view the full text, please login as a subscribed user or purchase a subscription. Click here to view the full text on ScienceDirect. Fig. 1 Mean and 95%CI in ambulant and non-ambulant DMD boys for (A) respiratory function (FVC% and PEF%) in relation to age (B) Performance of the upper limb functional scale: total score and sub-domains in relation to age (C) MyoGrip and MyoPinch in % of predicted values for age.
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