Casey’s General Stores customers contributed $1.35 million in August to support the Muscular Dystrophy Association‘s 2017 Summer Camp, where children living with MD enjoy a week of fun and make new friends at no cost to their families. Since the fundraising campaign was launched in 2006, $9 million has been…
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Two non-profits that work on standards that can facilitate drug development have created a guide to help those doing Duchenne’s muscular dystrophy research. The two are Critical Path Institute (C-Path) and the Clinical Data Interchange Standards Consortium (CDISC). The resource they created is the Duchenne Muscular Dystrophy Therapeutic Area User…
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#NORDsummit – Despite Criticism, Orphan Drug Act Is Working to Advance Needed Treatments, FDA Says
As Congress begins debate this week to overhaul the U.S. tax code, lawmakers should leave the Orphan Drug Act (ODA) — and the tax incentives it offers pharmaceutical companies to develop therapies for rare diseases — off the table. That’s the message being pushed by the National Organization for…
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Researchers Encouraged by Early Effectiveness, Safety Profile of Tricyclo-DNA in DMD Animal Study
Tricyclo-DNA (tcDNA), a class of therapeutics known as antisense oligonucleotides (AONs), has shown an important degree of effectiveness and a promising safety profile in a preclinical study with mice. The goal of the study is to test tcDNA’s potential use in treating Duchenne Muscular Dystrophy (DMD). The study, “Efficacy and…
The newly approved Duchenne muscular dystrophy therapy Exondys 51 (eteplirsen) stays in immune cells several days after leaving the blood, continuing to help regenerate muscle fibers, according to a study in mice. Researchers at the Children’s National Health System in the Washington area said the findings on immune cells known as…
The biotechnology company Capricor Therapeutics recently presented data that support the use of its peripheral intravenous delivery method in the upcoming HOPE-2 clinical trial testing the investigational agent CAP-1002 as a potential therapy for Duchenne muscular dystrophy (DMD). The data were presented during a poster session at the Alliance…
A new version of the CRISPR-Cas9 gene-editing technology called CRISPR-Gold has successfully restored the correct sequence of the dystrophin gene in a mouse model of Duchenne muscular dystrophy (DMD), a new study revealed. Researchers found that an injection of CRISPR-Gold into DMD mice led to an 18-times-higher correction rate and…
Mice with Duchenne muscular dystrophy compensate for the weakness in their breathing muscles by sending stronger signals to the nerves controlling the diaphragm, according to scientists at the University College Cork in Ireland. Now it is important to examine whether the same mechanisms exist in human Duchenne patients, researchers…
Orphan drug development, rising healthcare costs, patient assistance programs and controversial right-to-try legislation are of paramount interest to the parents and caregivers of boys with Duchenne muscular dystrophy (DMD). “One of the chief challenges of 21st Century pediatric medicine is our continued inability to provide more help to children born with…
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Resolaris Improved Muscle Strength of Nearly Two-thirds of FSHD Patients, Phase 1/2 Trial Shows
Resolaris (ATYR1940) improved the muscle strength of nearly two-thirds of adolescents and young adults with early-onset facioscapulohumeral muscular dystrophy (FSHD), according to final results of a Phase 1/2 clinical trial. The therapy’s maker, aTyr Pharma, presented the findings at the 22nd International Annual Congress of the World Muscle Society (WMS) in Saint…
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