research Archives - Page 53 of 145

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New Research Identifies Protein Epitope Targeted in Kawasaki Disease Immune Response

Post author:Jessica Lynn
Post published:April 15, 2020
Post category:Kawasaki syndrome

According to new research from the Journal of Infectious Diseases, a new protein epitope has been discovered while testing the immune response of people with Kawasaki Disease. Kawasaki Disease, also…

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Utrophin for Patients with Duchenne Muscular Dystrophy

Post author:Jessica Lynn
Post published:April 15, 2020
Post category:Duchenne Muscular Dystrophy

As originally discussed in Muscular Dystrophy News Today, a recent study has discovered that increasing levels of utrophin in the body can improve muscle strength, form, and function for patients…

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Is It Safe to Anesthetize Young Patients with Batten Disease?

Post author:Jessica Lynn
Post published:April 15, 2020
Post category:Batten Disease

How will this affect my child? Will this diagnosis change the way my child can receive medical treatment? If an emergency arises, do I have the knowledge needed to…

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Sex and Age Discrimination Could be Impeding Scientific Research for Conditions Like Cushing Syndrome

Post author:Trudy Horsting
Post published:April 15, 2020
Post category:Cushing Syndrome Rare Disease

NICHD The NICHD is the The Eunice Kennedy Shriver National Institute of Child Health and Human Development, one of the National Institutes of Health (NIH). It is the center for…

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Considerations for Rare Disease Patient and Caregiver Centricity in Clinical Trials

Post author:James Moore
Post published:April 15, 2020
Post category:Rare Disease

According to a story from XTalks, pulling off a clinical trial testing a new drug in a scientifically rigorous manner is no simple feat. It requires the triangulation of a…

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Fetal Spina Bifida Surgery Shown to Restore Brain Structure

Post author:Jessica Lynn
Post published:April 14, 2020
Post category:Chiari Malformation Rare Disease Spina Bifida

According to a press release from the Mayo Clinic, new research findings show that in-utero neurological surgery for patients with spina bifida is more successful in strengthening brain structure…

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Study: Felty’s Syndrome Presents Differently in Pediatric Patients

Post author:James Moore
Post published:April 14, 2020
Post category:Felty's syndrome Juvenile idiopathic arthritis Neutropenia Rheumatoid Arthritis Thrombocytopenia

According to a study published in BMC Pediatrics, the rare disease known as Felty's syndrome is primarily known to affects adults between the ages of 50 and 70, but on very rare…

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Phase 2b Prader-Willi Syndrome Trial Ends in Disappointment

Post author:James Moore
Post published:April 13, 2020
Post category:Congenital Adrenal Hyperplasia Prader-Willi Syndrome

According to a story from Malvern Daily Record, the biopharmaceutical company Millendo Therapeutics, Inc. recently released data from its phase 2b clinical trial. This study was testing the company's experimental…

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Parkinson’s Disease: A New Study Focusing on Preventing the Loss of Dopamine Producing Neurons

Post author:Rose Duesterwald
Post published:April 9, 2020
Post category:Parkinson's Disease

The drug levodopa and most other therapies developed over the years for Parkinson's disease have been centered on correcting the lack of dopamine. Researchers acknowledge that the reason dopamine…

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Is Six Feet Enough Distance to Protect from Coronavirus Transmission?

Post author:James Moore
Post published:April 9, 2020
Post category:COVID-19

According to a story from People's Pharmacy, a dizzying array of advice related to minimizing spread or coronavirus/COVID-19 has barraged our senses over the last few weeks as the number…

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COVID-19: The Purpose of Ventilators, Why There’s a Shortage, and What’s Being Done

Post author:Trudy Horsting
Post published:April 9, 2020
Post category:COVID-19

Every day on the news we hear the pleas for more medical masks for doctors and nurses, more personal protective equipment, and more ventilators. We all know what masks are,…

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Study Discovers New Method to Predict Duchenne Muscular Dystrophy Progression

Post author:James Moore
Post published:April 9, 2020
Post category:Duchenne Muscular Dystrophy Muscular Dystrophy

According to a study published in the journal Neurology, a measure called the vastus lateralis fat fraction was found to be useful in predicting the age at which a patient living…

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Study Confirms Effectiveness of Gamma Knife Surgery for Intracanalicular Vestibular Schwannoma

Post author:James Moore
Post published:April 8, 2020
Post category:Neurofibromatosis Type 2 Schwannoma

According to a study published in January in the scientific journal Acta Neurologica Scandinavica, the use of gamma knife surgery as a treatment for a rare tumor called a vestibular schwannoma can…

Intrahepatic Cholestasis of Pregnancy and its Link to Non-Alcoholic Fatty Liver Disease

Post author:Jessica Lynn
Post published:April 8, 2020
Post category:Intrahepatic cholestasis

According to a study summarized in Gastroenterology Consultant, pregnant women with intrahepatic cholestasis of pregnancy are much more likely to also be diagnosed with nonalcoholic fatty liver disease. You can…

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Study Highlights the Psychological Challenges of Mitochondrial Diseases

Post author:James Moore
Post published:April 7, 2020
Post category:Mitochondrial Disease Rare Disease

A recent study published in the Orphanet Journal of Rare Diseases examines the psychological burden of mitochondrial diseases in children that are suspected of having some form of it but have yet…

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CCHS Study Highlights Value of Screening Based on Family History

Post author:James Moore
Post published:April 7, 2020
Post category:Congenital central hypoventilation syndrome Rare Disease

According to a story from Hindawi, a recent study focused on congenital central hypoventilation syndrome serves to bring to light the importance of genetic testing of close family members based…

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New Myelin Discovery has Implications for Multiple Sclerosis and Other Diseases

Post author:James Moore
Post published:April 6, 2020
Post category:Multiple Sclerosis

According to a story from EurekAlert!, a recent discovery related to myelin, a fatty acid that coats neurons, could have significant implications for diseases that impact it, such as multiple…

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Investigative Therapy for Homocystinuria has Received Rare Pediatric Disease Designation

Post author:Trudy Horsting
Post published:April 6, 2020
Post category:homocystinuria

What is Homocystinuria? Classical homocystinuria, or HCU, is a rare metabolic condition which its caused by a deficiency of the CBS enzyme. It results in high levels of homocysteine in…

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Researchers Have Found an Early Sign of Alzheimer’s Disease in DNA Modification

Post author:Rose Duesterwald
Post published:April 3, 2020
Post category:Alzheimer disease type 3 Alzheimer's disease without Neurofibrillary tangles

In February 2020, Epigenetics published results of a study reporting that researchers have found an early sign of Alzheimer’s disease in DNA modification (methylation) that had been overlooked. Patients…

Post Mortem Gene Therapy Suggests Hopeful Results for Parkinson’s Disease Treatment

Post author:Jessica Lynn
Post published:April 2, 2020
Post category:Parkinson's Disease

Research findings can sometimes come from unexpected places. For example, according to Parkinson's News Today, the benefits and shortcomings of an investigative gene therapy were explored through the post-mortem analysis…

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Newborn Screening Tests Help Catch Fabry Disease Before Irreversible Symptoms Begin

Post author:Sunniva Bean
Post published:April 1, 2020
Post category:Fabry Disease Rare Disease

As originally reported in Fabry Disease News, the potentially fatal genetic disorder Fabry disease can be diagnosed most effectively in infancy by newborn screening programs. By identifying the disease early,…

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Two Part Treatment Proven Superior for Pulmonary Arterial Hypertension

Post author:James Moore
Post published:April 1, 2020
Post category:pulmonary arterial hypertension Systemic Sclerosis

According to a story from Pulmonary Hypertension News, the results of a recent phase 3 clinical trial have determined that patients with connective tissue disease-associated pulmonary arterial hypertension (CTD-PAH) stand…

Zolgensma for Spinal Muscular Atrophy Now Approved in Japan

Post author:Trudy Horsting
Post published:March 27, 2020
Post category:Spinal Muscular Atrophy

In May of 2019, the FDA approved a new therapy for Spinal Muscular Atrophy (SMA) called Zolgensma. Now, that therapy has been approved by the Japanese Ministry of Health, Labour…

Despite Advancements in Precision Medicine, We Also Need to Look at Disease Broadly

Post author:Trudy Horsting
Post published:March 27, 2020
Post category:Lupus Multiple Sclerosis Rheumatoid Arthritis

Published in Social Science & Medicine, a recent study conducted by Drexel University has shed light on how, even in a world of evolving precision medicine, there is also merit in…

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Unique Stem Cell Therapy Could Help Treat Gastroparesis

Post author:James Moore
Post published:March 26, 2020
Post category:Gastroparesis Rare Disease

According to a story from BioPorfolio, a recent study first published in STEM CELLS Translational Medicine describes a unique stem cell procedure that has the potential to be a major step…

New Research Identifies Protein Epitope Targeted in Kawasaki Disease Immune Response

Utrophin for Patients with Duchenne Muscular Dystrophy

Is It Safe to Anesthetize Young Patients with Batten Disease?

Sex and Age Discrimination Could be Impeding Scientific Research for Conditions Like Cushing Syndrome

Considerations for Rare Disease Patient and Caregiver Centricity in Clinical Trials

Fetal Spina Bifida Surgery Shown to Restore Brain Structure

Study: Felty’s Syndrome Presents Differently in Pediatric Patients

Phase 2b Prader-Willi Syndrome Trial Ends in Disappointment

Parkinson’s Disease: A New Study Focusing on Preventing the Loss of Dopamine Producing Neurons

Is Six Feet Enough Distance to Protect from Coronavirus Transmission?

COVID-19: The Purpose of Ventilators, Why There’s a Shortage, and What’s Being Done

Study Discovers New Method to Predict Duchenne Muscular Dystrophy Progression

Study Confirms Effectiveness of Gamma Knife Surgery for Intracanalicular Vestibular Schwannoma

Intrahepatic Cholestasis of Pregnancy and its Link to Non-Alcoholic Fatty Liver Disease

Study Highlights the Psychological Challenges of Mitochondrial Diseases

CCHS Study Highlights Value of Screening Based on Family History

New Myelin Discovery has Implications for Multiple Sclerosis and Other Diseases

Investigative Therapy for Homocystinuria has Received Rare Pediatric Disease Designation

Researchers Have Found an Early Sign of Alzheimer’s Disease in DNA Modification

Post Mortem Gene Therapy Suggests Hopeful Results for Parkinson’s Disease Treatment

Newborn Screening Tests Help Catch Fabry Disease Before Irreversible Symptoms Begin

Two Part Treatment Proven Superior for Pulmonary Arterial Hypertension

Zolgensma for Spinal Muscular Atrophy Now Approved in Japan

Despite Advancements in Precision Medicine, We Also Need to Look at Disease Broadly

Unique Stem Cell Therapy Could Help Treat Gastroparesis

The Let’s Chat CAR T One-on-One Mentor Program: Speaking with Someone Who Understands What You Are Going Through

“Shades of Psoriasis”: Plaque Psoriasis and Sharing Your Story

Understanding RSV: Why Combatting Ethnic and Age-Related Health Disparities Could Improve Outcomes

We Need Equity and Diversity Within Rare Disease Research

DNA in Color: How Ronya Nelson is Working to Close the Diversity Gap in Genetics - Part 1

Bridging the Diversity Gap in Healthcare

Congratulations to the 2023 Recipients of the Diversity in Lupus Research Awards!

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