The first patient has been dosed in a Phase 1/2 clinical trial of AMT-162, the one-time gene therapy in development by uniQure for treating amyotrophic lateral sclerosis (ALS) caused by mutations in the SOD1 gene, when the condition is thus known as SOD1-ALS. The open-label EPISOD1 clinical…
First SOD1-ALS patient dosed in trial of AMT-162 gene therapy
It was six years ago last week that my late husband, Jeff, and I sat in a neurologist’s office in Maryland as the doctor said, “I believe that this is ALS.” Moments before, I’d been watching the doctor administer a second electromyography (EMG) on Jeff, who found the procedure…
An ultra-high dose of mecobalamin, a naturally occurring form of vitamin B12, has been approved in Japan under the brand name Rozebalamin to treat amyotrophic lateral sclerosis (ALS). The therapy, which will be manufactured and marketed by Eisai, is intended to slow functional decline in people with the…
The Scott-Morgan Foundation (SMF), a nonprofit that’s pioneering assistive technology innovation, will lead a new initiative using artificial intelligence (AI) technologies to help people with diseases like amyotrophic lateral sclerosis (ALS) to better communicate and maintain their sense of identity. “We live in a world where millions are…
I’m thankful for the way technology is developing to improve the quality of life for people with ALS, including my husband, Todd. Six years ago, I encouraged Todd to bank his voice while it was still strong. He signed up with one service and went to work recording various…
A Texas Children’s Hospital investigator has received a $2.4 million grant for a study that seeks to understand whether mechanisms used by bacteria to hide from the immune system can be explored to ease inflammation in amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. Steven Boeynaems, PhD, an investigator…
A treatment candidate from Revalesio named RNS60 protected motor neurons from damage in a mouse model of amyotrophic lateral sclerosis (ALS) linked with accumulating toxic TDP-43 protein clumps. It also showed it could improve the health of mitochondria, the cells’ powerhouses that are abnormal in ALS, and reduce…
NeuroSense Therapeutics has initiated the process of applying to Canada’s regulators for early marketing approval of PrimeC in treating amyotrophic lateral sclerosis (ALS), based on positive findings in a Phase 2b clinical trial. The application will be filed under Health Canada’s Notice of Compliance with Conditions policy, which could…
Avextra is set to launch a Phase 2 trial in Italy to test a cannabis-based oral medication for the management of symptoms in people with amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. The announcement comes after clearance from both the Italian Medicines Agency and the Ministry of…
I like the Maroon 5 song “Memories” because I’m soothed by the melody, borrowed from Johann Pachelbel’s Canon in D, and I’m moved by the lyrics. One line in particular resonates with me: “The memories bring back you.” Though my husband, Todd, is still with me,…
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