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Article
Open AccessThe parent and family impact of CLN3 disease: an observational survey-based study
CLN3 disease (also known as CLN3 Batten disease or Juvenile Neuronal Ceroid Lipofuscinosis) is a rare pediatric neurodegenerative disorder caused by biallelic mutations in CLN3. While extensive efforts have been ...
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Article
Open AccessThree immunizations with Novavax’s protein vaccines increase antibody breadth and provide durable protection from SARS-CoV-2
The immune responses to Novavax’s licensed NVX-CoV2373 nanoparticle Spike protein vaccine against SARS-CoV-2 remain incompletely understood. Here, we show in rhesus macaques that immunization with Matrix-MTM adju...
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Article
Open AccessXBB.1.5 spike protein COVID-19 vaccine induces broadly neutralizing and cellular immune responses against EG.5.1 and emerging XBB variants
Monovalent SARS-CoV-2 Prototype (Wuhan-Hu-1) and bivalent (Prototype + BA.4/5) COVID-19 vaccines have demonstrated a waning of vaccine-mediated immunity highlighted by lower neutralizing antibody responses aga...
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Article
Open AccessImmunogenicity and protection of a variant nanoparticle vaccine that confers broad neutralization against SARS-CoV-2 variants
SARS-CoV-2 variants have emerged with elevated transmission and a higher risk of infection for vaccinated individuals. We demonstrate that a recombinant prefusion-stabilized spike (rS) protein vaccine based on...
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Article
Open AccessSARS-CoV-2 spike glycoprotein vaccine candidate NVX-CoV2373 immunogenicity in baboons and protection in mice
The COVID-19 pandemic continues to spread throughout the world with an urgent need for a safe and protective vaccine to effectuate herd protection and control the spread of SARS-CoV-2. Here, we report the deve...
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Article
Open AccessThe challenges of living with and managing epidermolysis bullosa: insights from patients and caregivers
Little information is available regarding the burden of living with and managing epidermolysis bullosa, including the distinct challenges faced by patients with different disease types/subtypes.
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Article
Open AccessRegulatory strategies for rare diseases under current global regulatory statutes: a discussion with stakeholders
Rare or orphan diseases often are inherited and overwhelmingly affect children. Many of these diseases have no treatments, are incurable, and have a devastating impact on patients and their families. Regulator...
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Article
Correction to: A Semi-Physiologically Based Pharmacokinetic Model Describing the Altered Metabolism of Midazolam Due to Inflammation in Mice
One of the authors has his name incorrectly indexed in PubMed and SpringerLink as “Laird Forrest M” (last name “Laird Forrest”). His name should index as “Forrest M. Laird” with last name as “Forrest”.
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Article
Open AccessEye Care Quality and Accessibility Improvement in the Community (EQUALITY) for adults at risk for glaucoma: study rationale and design
Primary open angle glaucoma is a chronic, progressive eye disease that is the leading cause of blindness among African Americans. Glaucoma progresses more rapidly and appears about 10 years earlier in African ...
