New England Journal of Medicine (NEJM) Announces Results of A Unique Study of Hirschsprung Disease Affecting Infants

  An article in BioSpace reports new information published online by the NEJM concerning Hirschsprung disease. The disease involves the absence of nerves in parts of the intestines prior to birth. Researchers…

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Biomarker Identified Which Can Determine Severity of Idiopathic Pulmonary Fibrosis

Nigam Shah and Purvesh Khatri, professors at Stanford University, along with graduate student Madeleine Scott have just announced results from a research study which could be pivotal for those living…

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The First Adeno-Associated Viral Gene Therapy for Huntington’s Disease Receives the FDA’s Fast Track Designation
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The First Adeno-Associated Viral Gene Therapy for Huntington’s Disease Receives the FDA’s Fast Track Designation

  A recent article in globenewswire.com news reported that the FDA has approved Fast Track Designation for AMT 130, an experimental gene therapy for the treatment of Huntington’s disease. The…

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Gene Therapy Could Change Rare Disease Treatment Forever, But Will Anyone be Able to Afford It?
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Gene Therapy Could Change Rare Disease Treatment Forever, But Will Anyone be Able to Afford It?

According to a story from BNN Bloomberg, gene therapies are without a doubt an upcoming advancement in care and treatment for many diseases that have a genetic origin. This includes…

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Could a “Gene Expression Atlas” Unlock the Secrets of Amyotrophic Lateral Sclerosis?

According to a story from MD Magazine, the team at Spatial Transcriptomics has utilized an innovative computational approach in order to map gene expression on the spinal cords of patients…

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Enrollment Completed in Achromatopsia and Retinitis Pigmentosa Clinical Trials

ACHM and XLRP Achromatopsia (ACHM) is a rare, inherited retinal disease which causes impaired cone photoreceptor function. Patients experience light sensitivity so extreme that it can cause blindness during the…

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Pulmonary Hypertension Drugs are Effective for Some Sarcoidosis Patients, Study Says
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Pulmonary Hypertension Drugs are Effective for Some Sarcoidosis Patients, Study Says

According to a story from Pulmonary Hypertension News, many patients with the rare disease sarcoidosis frequently experience lung complications. Included in these complications is pulmonary hypertension, a condition of elevated…

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Exciting Gene Therapy Progress in Huntington’s Disease and Spinocerebellar Ataxia

Huntington's Disease & SCAs  Huntington's disease (HD) and spinocerebellar ataxia (SCA) are both neurodegenerative diseases caused by the same type of error in the body's DNA. Essentially, the CAG or…

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Company Announces Plans to File for Conditional Marketing for Duchenne Muscular Dystrophy Drug in the EU

According to a story from Market Screener, the specialty pharmaceutical company Santhera Pharmaceuticals recently released an announcement in which the company revealed its intent to file for Conditional Marketing Authorization…

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