According to a story from globenewswire.com, the pharmaceutical company Novartis recently announced that its CAR-T cell therapy drug Kymriah recently gained approval from the European Commission. The drug was approved…
A recent study suggests an alternative procedure for delivering the drug nusinersen to patients being treated for spinal muscular atrophy. Although more research needs to be done to estabilish its…
Two clinical trials have had their study protocols amended, announces Pfizer and Astellas Pharma. The Phase 3 trials, called ‘Arches’ and ‘Embark’, will investigate the safety and effectiveness of the…
Tetra Bio-Pharma has submitted several Orphan Drug Designation applications to the US Food and Drug Administration. The applications are on behalf of cannabinoids for the treatment of multiple rare forms…
According to a story from Akcea Therapeutics, the company recently received a reply from the US Food and Drug Administration (FDA) in regards to their New Drug Application for WAYLIVRA…
For comedy and Saturday Night Live fans, Pete Davidson needs no introduction. For those out of the loop, you might now be hearing more of this young comedian who is…
On August 4th, the Ehlers-Danlos Society led the Zebra Strong Rally, which concluded the third day of the Ehlers-Danlos Syndrome Learning Conference in Baltimore, Maryland. Advocating for Ehlers-Danlos Syndrome The…
A group of researchers studying the causes underlying myelodysplastic syndromes have found that HIF1A may play an important role in these conditions. For more detailed information, you can read the…
A recent study published in the journal Nature Communications highlights a potential therapy for the treatment of muscular dytrophies and other diseases linked to mutations of the FKRP gene. In the study, the…
According to a story from BioSpace, the biopharmaceutical company AbbVie recently announced that the US Food and Drug Administration (FDA) has approved a new combination treatment for Waldenstrom's macroglobulinemia (WM).…
According to a story from globnewswire.com, the gene therapy company Adverum Biotechnologies recently announced that the US Food and Drug Administration (FDA) has granted them Orphan Drug designation for their…
According to a story from Medical Xpress, a team of scientists from Michigan State University have managed to reveal the manner in which a genetic malfunction can lead to the…
The National Institutes of Health (NIH) have granted the University of Alabama at Birmingham $8.9 million to fund research into an investigational drug for idiopathic pulmonary fibrosis. You can read…
An article by the Evening Standard announces new research towards a cure for cases of genetic blindness. The doctors pursuing this research are able to develop specific parts of the…
The drug minocycline is a tetracycline antibiotic. Previous research using mouse models has indicated that it may have anti-inflammatory and antiapoptotic (prevents cell death) properties and may benefit some neurological…
According to Digital Health Age, a wearable eye technology may be revolutionary in the lives of those who have Locked-In Syndrome, which is often associated with ALS and other motor…
Two companies, Tolero Pharmaceuticals Inc. and AbbieVie, have agreed to collaborate to clinically research a potential combination therapy for relapsed or refractory acute myeloid leukaemia. The potential therapy is comprised…
The investigational drug AT132 has been awarded Regenerative Medicine Advanced Therapy designation for the treatment of X-linked Myotubular Myopathy by the United States FDA. You can read the source press…
Two studies have looked into the long-term effects of subthalamic deep brain stimulation as a treatment for Parkinson’s disease. For more detailed information on this topic, you can find the…
According to Newswise, new findings indicate that electrical stimulation of the spinal cord and brain may be effective in treating symptoms of a group of rare neurological movement disorders, neurodegenerative ataxias.…
August is Stevens-Johnson Syndrome (SJS) Awareness Month! In honor of the SJS community, Friday August 31st has been designated an awareness day. What is Stevens-Johnson Syndrome? SJS is a rare, serious…
Unknown origins of birth. Overcoming enormous personal obstacles at an early age. A mysterious medical diagnosis. Making a drastic new change in life. This could - and is! - a…
The speciality pharmaceutical company Tikomed has announced that the UK Medicines and Healthcare Products Regulatory Agency has granted the company permission to go ahead with a Phase 2 clinical study.…
I came into the hospital about four days ago at 6 am in extreme abdominal pain. I remember writhing on the emergency room bed and breathing so frantically that my…
According to a story from BioSpace, the US Food and Drug Administration (FDA) recently approved the drug Oxervate (cenegermin). This treatment is the first FDA approved therapy for the rare…
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