NORWELL, Mass. — The National Organization for Rare Disorders (NORD®) today announced the addition of seven leading academic medical and research centers to its national NORD® Rare Disease Centers of Excellence Network, expanding collaboration to improve care and accelerate research for the more than 30 million Americans living with rare conditions. With...
treatment News
Allschwil, Switzerland – Idorsia Ltd (SIX: IDIA) announces the publication of results from the pivotal Phase 3 MODIFY study and its open-label extension (OLE) evaluating lucerastat, an oral substrate reduction therapy, in adults with Fabry disease. The data, published in Nature Communications, titled “Lucerastat, an oral therapy for Fabry disease: Results...
TORONTO, Canada – Satellos Bioscience Inc. (TSX: MSCL, OTCQB: MSCLF) (“Satellos” or the “Company”), a clinical-stage biotechnology company developing life-improving medicines to treat degenerative muscle diseases, today announced the publication in Nature Communications of new research from a scientific team at the Ottawa Hospital Research Institute (OHRI) that validates the company’s novel...
NAARDEN, Netherlands, WALTHAM, Mass. & BARCELONA, Spain — Prilenia Therapeutics B.V. and Ferrer today announced the publication, in the journal Nature Medicine, of a manuscript entitled “Pridopidine in Early-Stage Manifest Huntington Disease: A Phase 3 Trial“ii. The publication describes data showing that treatment with pridopidine slowed clinical progression in Huntington’s disease (HD)...
CAMBRIDGE, Mass. — Biogen Inc. (Nasdaq: BIIB) today announced that Nature Medicine published results from the Phase 2/3 DEVOTE study evaluating the high-dose regimen of nusinersen, comprised of 50 mg/5 mL loading and 28 mg/5 mL maintenance doses, in spinal muscular atrophy (SMA). The high-dose regimen of nusinersen offers a more rapid loading...
CHENGDU, China — Sichuan Kelun-Biotech Biopharmaceutical Co., Ltd. (“Kelun-Biotech”, HKEX: 6990) announced that clinical data from a Phase II study evaluating novel TROP2 antibody-drug conjugate (ADC) sacituzumab tirumotecan (sac-TMT) in combination with PD-L1 monoclonal antibody (mAb) tagitanlimab for the first-line treatment of advanced or metastatic non-small cell lung cancer (NSCLC) have been...
LAS VEGAS — Bone sarcoma is a rare and aggressive form of cancer that originates in the bone tissue. It poses significant challenges during its treatment due to several factors. Firstly, its rarity often leads to delayed diagnosis, as symptoms can mimic other common conditions. Additionally, bone sarcoma typically affects...
Bethesda, Maryland – Researchers have shown that dangerous cysts, which form over time in polycystic kidney disease (PKD), can be prevented by a single normal copy of a defective gene. This means the potential exists that scientists could one day tailor a gene therapy to treat the disease. They also...
NEW YORK, NY — Syndax Pharmaceuticals (Nasdaq: SNDX), a commercial-stage biopharmaceutical company advancing innovative cancer therapies, today announced that the National Comprehensive Cancer Network® (NCCN®) Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Acute Myeloid Leukemia were updated to include revumenib as a category 2A recommendation for relapsed or refractory (R/R)...
PLYMOUTH MEETING, PA — The National Comprehensive Cancer Network® (NCCN®) is announcing several new updates to the cancer treatment practices listed in the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®). The evidence-based expert consensus recommendations are the most frequently updated guidelines for any area of medicine and are used...