SOUTH SAN FRANCISCO, Calif. – Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that Evrysdi™ (risdiplam) data from the dose finding Part 1 of the pivotal FIREFISH study in infants with symptomatic Type 1 spinal muscular atrophy (SMA) were published in the February 24,...
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Systemic sclerosis (SSc) is a rare disease that impacts up to 75,000 people in the United States Approximately 80% of SSc patients may be affected by interstitial lung disease (ILD), a progressive disease that can significantly impact lung function and can be life-threatening In a global study, Actemra reduced the...
SOUTH SAN FRANCISCO, Calif. — Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today data from the Phase III OCARINA II study (S31.006) of Ocrevus® (ocrelizumab), an investigational twice-yearly, 10-minute subcutaneous (SC) injection. Results showed near-complete suppression of clinical relapses and brain lesions in people...
These indigenous products will lead to drug prices for rare diseases being slashed up to 100 times of their current market value CHENNAI, India – Providing relief to patients with rare diseases across India, the Union Health Ministry has made available generic drugs to support the care and treatment of...
PARIS, France — Genethon, a non-profit research organization focused on developing gene therapies for rare diseases, today marks International Rare Disease Day by highlighting its efforts to bring gene therapies to patients suffering from rare diseases such as limb girdle muscular dystrophies, Crigler Najjar syndrome and Duchenne muscular dystrophy. Globally...
BUFFALO, NY – A new research perspective was published in Oncotarget, entitled, “Genetic alterations in thyroid cancer mediating both resistance to BRAF inhibition and anaplastic transformation.” In this new paper, researchers Mark Lee and Luc GT Morris from New York Presbyterian Hospital and Memorial Sloan Kettering Cancer Center discuss thyroid...
BUFFALO, NY – A new research paper was published in Oncotarget’s Volume 15 entitled “Genetic and therapeutic landscapes in cohort of pancreatic adenocarcinomas: next-generation sequencing and machine learning for full tumor exome analysis.” About 7% of all cancer deaths are caused by pancreatic cancer (PCa). PCa is known for its...
Toronto, Ontario – A Canadian-led study has identified genes which may be partially responsible for the development of cerebral palsy. Cerebral palsy (CP), a condition that affects the development of motor skills in children, is the most common childhood-onset physical disability. CP can have different causes, such as infections, injuries,...
Scientists have found genetic variations that appear to increase the risk of developing primary biliary cirrhosis, a baffling disease that can destroy the liver. Primary biliary cirrhosis is an autoimmune disorder that blocks the bile ducts in the liver. It is a condition that mostly affects women, striking about one...
A team led by Nagoya University Graduate School of Medicine in Japan has investigated screen time in autistic (ASD) and ADHD children. Screen time refers to the amount of time a person spends on a device with a screen, such as a smartphone, computer, television, or video game system. The...