Toronto, Canada – Researchers at The Hospital for Sick Children (SickKids) have found that inflammation in an immune cell may be responsible in part for some severe symptoms in a group of rare genetic conditions called lysosomal storage diseases (LSDs). LSDs affect about one in 7,700 live births worldwide. Children...
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TORONTO, Canada – A new round of clinical trials supported by the Ontario Institute for Cancer Research (OICR) will harness a unique opportunity to test some of the newest treatment strategies for a range of different cancers. OICR has announced two new clinical trials as part of its Window-of-Opportunity (WOO)...
SOUTH SAN FRANCISCO, Calif. – Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced new exploratory 2-year longer-term data from Part 2 of SUNFISH, a global placebo-controlled study evaluating Evrysdi™ (risdiplam) in people aged 2-25 years with Type 2 or non-ambulant Type 3 spinal muscular...
COPENHAGEN, Denmark — Ascendis Pharma A/S (Nasdaq: ASND) today announced new data from Week 214 of its Phase 2 PaTH Forward Trial showing that long-term treatment with TransCon PTH (palopegteriparatide) continued to provide a durable response in adults with hypoparathyroidism. Results were shared in an oral presentation today by Andrea...
New Rochelle, NY – Scientists from Spirovant Sciences describe SP-101, a novel adeno-associated virus (AAV) gene therapy for the treatment of cystic fibrosis (CF) in the peer-reviewed journal Human Gene Therapy. They also showed that after single dose inhaled delivery of SP-101, SP-101 vector genomes were detected throughout the respiratory tract...
Barcelona, Spain – Researchers at the Centre for Genomic Regulation (CRG), the University of the Basque Country (UPV/EHU), Donostia International Physics Center (DIPC) and the Fundación Biofisica Bizkaia (FBB, located in Biofisika Institute) have developed an artificial intelligence which can differentiate cancer cells from normal cells, as well as detect...
LINKÖPING, Sweden – AMRA Medical is pleased to announce the publication of new research in Neurology demonstrating the potential of quantitative whole-body MRI to measure disease progression in Spinal and Bulbar Muscular Atrophy (SBMA), a rare, progressive neuromuscular disease. The research was also selected for the cover of the issue, providing...
Ridgefield, Connecticut – Boehringer Ingelheim announced new analyses to be presented at the American Academy of Dermatology (AAD) annual meeting in Orlando that explore the effect that SPEVIGO® (spesolimab-sbzo) may have on the physical symptoms and mental health burden associated with generalized pustular psoriasis (GPP). These post-hoc exploratory analyses from the...
DUBLIN – Horizon Therapeutics plc (Nasdaq: HZNP) today announced the publication of a new analysis of the pivotal Phase 2/3 N-MOmentum trial for UPLIZNA (inebilizumab-cdon), assessing the potential for reduced risk of worsening disability in those living with NMOSD. These data are published in the May issue of Neurology Neuroimmunology...
An exposure-outcome analysis in 72 patients with refractory non-small cell lung cancer (NSCLC) who received AFM24 at 480 mg weekly demonstrates that higher drug exposure (above median) leads to improved objective response rate (33.3% vs 5.6%) and longer progression free survival (PFS) (7.3 mo. vs 2.9 mo.) without a...