CRISPR has seen a number of developments recently; 2020 was a good year for gene therapy. Medical professionals are excited by the improvements made and motivated to make further discoveries.…
Science 37 and Xperiome have entered into a partnership in order to allow for more viable rare disease research, according to an article in PR Newswire. If the partnership goes…
A report, commissioned by the National Organization for Rare Disorders and released by IQVIA, has revealed that the majority of orphan drugs, 79%, treat only rare diseases. Despite this fact,…
Living with a rare disease can be difficult and scary for some, especially during a global pandemic that has affected millions. This Nottingham father knows these feelings firsthand; he lives…
Being diagnosed with a rare disease can bring on various emotions; everybody's experience is unique to them. Some feel relieved to finally put a name to their symptoms; others are…
Pharnext has released an update on the clinical development of PXT3003, a treatment for Charcot-Marie-Tooth disease type 1A (CMT1A). According to the biopharmaceutical company, they are on track to dose…
Parker Boyer and his family know many of the obstacles that rare disease patients face; they've encountered many of them since Parker's birth. In fact, they dealt with a number…
Evotec SE and Chinook Therapeutics have entered into a strategic partnership in an effort to find, create, and develop viable treatments for those living with chronic kidney diseases. Under the…
Together, Takeda Pharmaceuticals and the Children's National Hospital have created a program that aims to create and improve a standard of care for rare disease patients. As these patients often…
According to BioSpace, Kronos Bio will continue with its plans for a Phase 3 trial of entospletinib after a positive meeting with the FDA. One of the meeting's focuses was…
Researchers from Northwestern University have discovered a compound with the ability to stop the progressive degeneration of motor neurons in those with amyotrophic lateral sclerosis (ALS). The continued breakdown of…
Like many other residents of West Hollywood, Mark Chaney uses public transportation, specifically the bus. Twice a day, Monday through Friday, Mark boards the bus at his local stop. He…
Monty Hui, an eight-year-old boy from Australia, and his family have spoken to SBS News about living with a rare disease and the obstacles faced in diagnosis and recognition. He…
There's positive news for Evrysdi, a treatment for spinal muscular atrophy (SMA). The EMA Committee for Medicinal Products for Human Use (CHMP) has recommended that the drug be approved, and…
Pharnext has announced that they will soon open enrollment for their PREMIER trial of PXT3003, a Charcot-Marie-Tooth disease type 1A (CMT1A) treatment. Beginning in March, researchers will search for 350…
Researchers from the Universities of Nottingham and Exeter have discovered a new, possible treatment method for Duchenne muscular dystrophy (DMD). Their research demonstrated that treating hydrogen sulfide (H2S) deficiency in…
It's been almost a full year since the beginning of the COVID-19 pandemic, and we've learned a lot about the novel coronavirus since then. For one, we now know that…
According to GlobeNewswire, Tiziana Life Sciences and Parexel Biotech have agreed to collaborate on a Phase 1b/2 trial of foralumab, an oral treatment for Crohn's disease. As previous research has…
Julia's Wings Foundation is gearing up for the Operation Wear Red Campaign in an effort to raise awareness for the rare blood disorder aplastic anemia. From the first of March…
The Brazilian Health Regulatory Agency, ANVISA, has granted market approval to Insightec for their focused ultrasound, Exablate 4000. This MR-guided ultrasound is used for the treatment of tremors that define…
According to BioSpace, Biohaven Pharma has utilized their MATE platform to create and develop a hyperimmune globulin mimic (HGM) that is able to both bind to and neutralize various strains…
Michael Conway, a grandfather from Scotland, believes that there should be increased rare disease screening after he was diagnosed with adrenoleukodystrophy (ALD) in 2016. Like many other rare disease patients,…
The Cystic Fibrosis Foundation is dedicated to finding a cure for the rare, genetic disorder. They fund more CF research than any other organization, and their latest effort is a…
The FDA has recently accepted NeoImmuneTech's Investigational New Drug (IND) application for NT-17, a treatment for progressive multifocal leukoencephalopathy (PML). Now that the application has been cleared, the biopharmaceutical company…
From February 25-27, the Americas Committee for Treatment and Research in Multiple Sclerosis will run its ACRTIMS Forum 2021, according to BioSpace. At this forum, Clene Inc. will present interim…
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