According to a story from UNC Health Care, the Belgian drug company Argenx recently announced that its investigational treatment efgartigimod has performed well in a phase 3 clinical trial. The…
Many myasthenia gravis patients do not benefit from already existing therapies, such as steroids, acetylcholinesterase inhibitors, or immunosuppressants. It is this fact that has pushed many biotechnology companies to start…
In a recent article that appeared in Science Magazine, Amy Boland described her twelve-year quest to find a cure for the cancer that invaded her lymph system. At first,…
Because of COVID-19, many health and medical-related conferences and events have been moved to virtual settings. But that doesn't mean that the learning has stopped, says Healio. During the Heart…
Immune-Onc Therapeutics recently received clearance from the FDA to begin trials of IO-202, a treatment for acute myeloid leukemia (AML). This news comes after the cancer immunotherapy company filed an…
The European Commission has recently granted approval to Bristol Myers Squibb (BMS) for their multiple sclerosis (MS) treatment, Zeposia. It has already been approved by the FDA in the US…
Medical professionals are already aware that inhaling air pollution can increase one's risk of a number of conditions, including respiratory and heart diseases. They are now discovering that it can…
Libtayo, a product of Regeneron Pharmaceuticals, has shown positive results in the treatment of those with advanced cutaneous squamous cell carcinoma. This data comes from a Phase 2 trial and…
If you think about it, our brains are pretty amazing. It is one of our most complex features and contains tens of billions of neurons which help control the…
HDSA 35 New Orleans: The 35th Annual HDSA Convention June 4th-6th, 2020 The 35th annual convention is tailored for Huntington's disease patients and their families. Join the HDSA for three…
Parent Project Muscular Dystrophy is a nonprofit that aims to find a cure for Duchenne muscular dystrophy. One of their programs, the Certified Duchenne Care Center Program (CDCCP), has recently…
In high school, Chuck Graham loved sports. He was a student-athlete dedicated to his teams. But then, at 16 years old, a car accident paralyzed Graham from the chest…
CytRx Corporation has recently received the Fast Track designation for their ALS treatment, arimoclomol. This status was also given for the treatment of Niemann-Pick disease and sporadic Inclusion Body Myositis…
According to a story from Medical Xpress, an Italian study has found that people in urban areas are at a 29 percent greater risk of developing multiple sclerosis (MS). This…
What would motivate a man, who has been living with amyotrophic lateral sclerosis (ALS) for twenty years, to accept an invitation to become part of a five-man team and…
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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…
The events of the past week-- and the past few hundred years-- have brought up a lot of sadness, anger, and reckoning in our country. There are so many ways…
In an article in Medical Xpress, Japanese doctors have performed a “first” by transplanting liver cells (made with embryonic stem cells) into a newborn. The six-day-old infant was diagnosed with…
RARE on the Road: a Rare Disease Leadership Tour March 28th, 2020 Minneapolis, MN The Rare Disease Leadership Tour is designed to give insights and education to rare disease advocates,…
According to a story from prnewswire.com, the Chiesi Global Rare Diseases and the biopharmaceutical company ProTalix Biotherapeutics have recently announced that they have submitted a Biologics License Application (BLA) for…
By Danielle Bradshaw from In The Cloud Copy The cells of hemophilia A patients were taken and genetically modified so that they would create an active clotting factor VIII (or…
According to a story from Pulmonology Advisor, a recent study has evaluated risk factors for the appearance of bronchiectasis in patients with aspirin-exacerbated respiratory disease (AERD). A small number of…
For the last few months, we've been inundated with stories of COVID-19, and with good reason, considering the global pandemic reached over 5 million cases worldwide as of this…
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…
Jalee Pelissier lives with muscular dystrophy. When she was just 13 she became an advocate for others with disorders or disabilities like her own. Now 20, her advocacy has only…
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