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Pivots During Pandemic Bring Patient-Centric Options to Rare Disease Patients


Published: Mar 01, 2021
By Heather McKenzie
February 28 marked Rare Disease Day, and biotech companies gathered together to raise awareness, advocate for improved access to treatment and let patients know how they are pivoting to improve patient care and access during the COVID-19 pandemic and beyond. 
Rezolute Inc., a clinical-stage biopharmaceutical company developing novel therapeutics for patients with metabolic and orphan diseases, was just beginning a Phase IIb study of its lead candidate, RZ358, for Congenital hyperinsulinism (HI), when the chaos struck.
“We have hospitals around the world where the COVID ward was right next to the research ward. We had hospitals that for six months shut down entirely,” shared Erin O’Boyle, Head of Clinical Operations at Rezolute. “We had a lot of overnight stays, but then, right when we were kicking off the study and going into enrollment and starting to activate our sites, COVID hit, and it really kind ....

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Sio Gene Therapies Announces First Patient Dosed in Clinical Trial of AXO-AAV-GM2 in Patients with Tay-Sachs and Sandhoff Disease (GM2 Gangliosidosis)


Published: Feb 03, 2021
 - First potentially disease-modifying gene therapy for GM2 gangliosidosis to enter clinical studies
- Expect to continue patient identification, screening, and enrollment in Stage 1 of the study throughout 2021
NEW YORK and RESEARCH TRIANGLE PARK, N.C., Feb. 03, 2021 (GLOBE NEWSWIRE) Sio Gene Therapies Inc. (NASDAQ: SIOX), a clinical-stage company focused on developing gene therapies to radically transform the lives of patients with neurodegenerative diseases, today announced that the first patient with infantile Tay-Sachs disease has been dosed in a Phase 1/2 trial evaluating AXO-AAV-GM2, an investigational gene therapy for the treatment of GM2 gangliosidosis, also known as Tay-Sachs or Sandhoff disease. ....

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Glioblastoma Multiforme (GBM) Treatments Advancing as FDA Steps Up Measures Due to Rising Incidences


Glioblastoma Multiforme (GBM) Treatments Advancing as FDA Steps Up Measures Due to Rising Incidences
PALM BEACH, Fla., Dec. 17, 2020 /PRNewswire/ Glioblastoma multiforme (GBM) is the most common and aggressive malignant brain tumor in humans that involves glial cells. GBM is present in two variants namely, giant cell glioblastoma and gliosarcoma. Gliomas are tumors arising from glial cells and may occur in the spinal cord or the brain, the latter being more common. Gliomas are the most common type of brain tumor and can be either supratentorial or infratentorial. Seizure, nausea, headache, hemiparesis and memory loss are some of the common symptoms observed in the patients suffering from GBM. GBM is most commonly found in male with the age of 65 years and above. Increasing habits of alcohol consumption, changing lifestyle and increasing incidence of seizure are some of the drivers for the growth of GBM market. GBM is generally treated by first surgically removing the tumor ....

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Best Penny Stocks To Buy For Under $4.50? 5 To Watch Today


Penny Stocks to Buy Under $4.50: Harmony Gold Mining Co. 
Harmony Gold Mining Co. is a major mining company working on the exploration, extraction and processing of gold in Africa. This includes mining operations in both South Africa and Papua New Guinea. In addition to mining gold, the company is quite diverse and mines uranium, silver and copper. In September, HMY posted its financial results for the third quarter of 2020. While the results were somewhat disappointing, it is understandable considering the effects of the covid pandemic on mining. Regardless, HMY stock has done quite well compared to many other companies throughout 2020. One of the things to note is that gold demand usually stays quite high during times of economic turmoil.  ....

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