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Anavex Life Sciences Corporation has recently announced that they will conduct a clinical trial for their small molecule, ANAVEX®3-71. This therapy is intended to treat neurodegenerative and neurodevelopmental conditions, such…
Passage Bio has been granted the Rare Pediatric Disease designation for their GM1 gangliosidosis treatment, PBGM01. This designation means that Passage will receive a priority review voucher, and it is…
A study has recently been conducted that suggests that rituximab may be preferable over traditional treatments for myasthenia gravis. It also concluded that tiuximab is the most effective if it…
Seelos Therapeutics has recently received the Rare Pediatric Disease Designation for their Sanfilippo syndrome treatment, trehalose. This designation is an incentive for companies to make therapies for conditions with an…
Loneliness is difficult for everyone regardless of the situation, but the current pandemic has definitely amplified it. In a time of isolation and distancing, it's difficult to not feel alone.…
Zolgensma, a gene therapy for spinal muscular atrophy (SMA), has recently been granted conditional approval by the European Commission (EC). This is extremely exciting news for those living with SMA…
It is no secret that COVID-19 has turned life as we know it upside down. It has changed everything from hanging out with friends to working to going grocery shopping.…
Enanta Pharmaceuticals has recently released the results from the second phase of its INTREPID trial, which studied EDP-305 as a treatment for primary biliary cholangitis (PBC). The results were positive,…
The understanding of rare diseases is often lacking, making it difficult for rare disease patients to get the diagnosis and treatment that they need. Dr. Tracy M. French aims to…
Protalix BioTherapeutics has recently announced the results from the third phase of the BRIDGE trial, which evaluated pegunigalsidase alfa for the treatment of Fabry disease. As these results were positive,…
Cytokine storms have been prominent in the news lately due to their role in COVID-19, but researchers have been studying their connection to Castleman disease for a long time. In…
The FDA has recently granted GTX-102, a treatment for Angelman syndrome, the Fast Track designation. This designation means that the review process is expedited. It is meant specifically for drugs…
The TACERN Study Group has recently conducted a study in order to discover an algorithm that detects tubers during MRIs. Deep learning was employed in an effort to apply this…
Axcella, a biotechnology company based out of Cambridge, Massachusetts, has released the results of AXA1125-003. According to Biotech 365, this study showed positive results for those with non-alcoholic fatty liver…
Researchers at the University of Houston have been investigating lupus nephritis and discovered that there are race-specific urinary biomarkers. Hugh Roy and his team have found that there are specific…
ProMIS Neurosciences is a biotechnology company that works specifically with antibody treatments, and they have recently developed intrabodies for the treatment of amyotrophic lateral sclerosis (ALS). This same therapy has…
Kubota Vision, a subsidiary of Kubota Pharmaceutical Holdings, has recently finished enrollment in the third phase of their emixustat hydrochloride (emixustat) trial. Emixustat hydrochloride is a treatment for Stargardt disease…
Phase III of the CheckMate -9ER trial has reached its primary endpoint of progression free survival in those with advanced or metastatic renal cell carcinoma. This study was evaluating CABOMETYX…
Maddie Nordhoy has a smile that can light up the room. She constantly brings joy to those around her, especially her family. In fact, she is even the inspiration for…
U.S. company, Vertex Pharmaceuticals, is the creator of three treatments for cystic fibrosis: Orkambi, Symdeco, und Kalydeco. These drugs were unavailable in Switzerland, as Vertex and Swiss health officials were…
The Connect MPS Patient Registry was created in 2016 to learn more about Sanfilippo syndrome and mucolipidoses (ML). 24 advocacy organizations are a part of this registry, all of which…
Fabry disease brings symptoms that can affect multiple organs throughout the body. The kidneys and gastrointestinal system are commonly affected, which can make life difficult. According to an article from…
Predicting the disease progression of idiopathic pulmonary fibrosis (IPF) is extremely important, as this condition progresses differently depending on the affected individual. Knowing if one has a poor outcome while…
Ibudilast is a drug that has been used to treat asthma, but it is now being evaluated as a treatment for COVID-19. Acute respiratory distress syndrome (ARDS) is a severe…
Due to the current COVID-19 pandemic, it is very difficult for people to visit their doctors. For some, this is not the biggest deal; they can stand to miss an…
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