Pratteln, Switzerland – Santhera Pharmaceuticals (SIX: SANN) announces that the China National Medical Products Administration (NMPA) has accepted for priority review the new drug application (NDA) for vamorolone in Duchenne muscular dystrophy (DMD) which was submitted by Sperogenix Therapeutics, Santhera’s specialized rare disease partner for China. The Center for Drug...
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Specific gene profiles of bronchoalveolar lavage (BAL) cells are associated with phenotypic manifestations of sarcoidosis such as activity and disease severity, including immune, inflammatory, and profibrotic mediators as identified via genome-wide transcriptome analysis, according to results published in the European Respiratory Journal. Study results also suggest 4 possible sarcoidosis endotypes with specific...
LANGHORNE, Pa — Savara Inc., a clinical stage biopharmaceutical company focused on rare respiratory diseases, recently launched aPAP ClearPath™, a new serum-based blood test that can be used by physicians in the U.S. to obtain a definitive diagnosis of aPAP, a rare autoimmune lung disease mediated by autoantibodies targeting GM-CSF....
Yokohama, Japan – Even though psychiatric disorders such as schizophrenia, bipolar disorder, and autism spectrum disorder (ASD) are quite common, their diagnosis and treatment are challenging. While doctors today have a good idea of the clinical symptoms caused by these disorders, our overall understanding of their biological characteristics and underlying...
CLINUVEL today announced that it has reached agreement with clinical and academic experts to expand its DNA Repair Program to patients diagnosed with xeroderma pigmentosum-variant (XP-V). The Program commenced in 2020, evaluating SCENESSE® (afamelanotide 16mg) in XP-C patients.“The progression of making afamelanotide available to a further group of XP patients, who...
AMSTERDAM, the Netherlands — Scenic Biotech, a pioneer in the discovery of genetic modifiers developing therapeutics to treat severe diseases, today announced positive preclinical data for its lead small molecule QPCTL inhibitor, SC-2882. The data was also presented today by Leandro Cerchietti, MD, Richard A. Stratton Associate Professor in Hematology...
Swindon, UK – Researchers have shown for the first time that a crucial interface in a protein that drives cancer growth could act as a target for more effective treatments. The study was led by the Science and Technology Facilities Council (STFC) Central Laser Facility (CLF) and used advanced laser...
BOSTON, Mass.c – In a paper published in the Journal of the American Medical Association (JAMA), neurologists at Beth Israel Deaconess Medical Center (BIDMC) showed that a simple skin biopsy test detects an abnormal form of alpha-synuclein, the pathological hallmark of Parkinson’s disease and the subgroup of neurodegenerative disorders known...
PROVIDENCE, R.I. — Agent Orange, an herbicide used during the Vietnam War, is a known toxin with wide-ranging health effects. Even though Agent Orange has not been used for decades, there is increasing interest in its effects on the brain health of aging veterans. A new study by scientists at...
Wilmington, DE — In a potential advance for melanoma patients, researchers at ChristianaCare’s Gene Editing Institute have used CRISPR gene editing tools to disable a gene mutation often seen in aggressive forms of this dangerous skin cancer that renders promising treatments ineffective. The study published in the CRISPR Journal focused...