Personalized ASO Provides Improvements for a Girl with KAND, an Ultra-rare Disease
Within the world of rare diseases exist conditions so sparse and infrequent they’re called nano-rare — or N of 1 — diseases and typically affect only one to thirty people worldwide (1). While less than 10% of rare diseases have approved treatments, nano-rare or ultra-rare diseases are often entirely excluded from drug development programs (1). However, the sphere of ...
Angelman Syndrome Therapies Show Positive Results in Early Phase Clinical Trials
A clinical trial for a drug designed to treat the rare neurodevelopmental disorder known as Angelman syndrome (AS) has achieved a favorable safety profile and lessening of overall symptoms in trial participants. In a recent press release, Ionis announced the detailed HALOS study results for the multiple ascending dose (MAD) portion of the Phase 1/2 open-label study of ION582. ...
Encouraging Progress in the Hunt for a Huntington’s Treatment
Described as a combination of Alzheimer's disease, Amyotrophic lateral sclerosis, and Parkinson's disease all at once, Huntington's disease is a rare and debilitating neurological disorder passed down within affected families. With a 50% chance of a child inheriting it from a parent with the HD mutation, the fatal disease often leaves a devastating family legacy. In the United States ...
Sequence- and Structure-Dependent Cytotoxicity of Phosphorothioate and 2′-O-Methyl Modified Single-Stranded Oligonucleotides
Authors: Laura V. Croft Mark Fisher, Tabassum Khair Barbhuiya, Serene El-Kamand, Samuel Beard, Aleksandra Rajapakse, Roland Gamsjaeger, Liza Cubeddu, Emma Bolderson, Ken O’Byrne , Derek Richard derek.richard@qut.edu.au, and Neha S. Gandhi Published Online: 17 June 2024 Abstract Single-stranded oligonucleotides (SSOs) are a rapidly expanding class of therapeutics that comprises antisense oligonucleotides, microRNAs, and aptamers, with ten clinically approved molecules. Chemical ...
Nucleic Acid Therapeutics: Successes, Milestones, and Upcoming Innovation
Authors: Jillian Belgrad, Hassan H. Fakih, and Anastasia Khvorova Published Online: 3 April 2024 Abstract Nucleic acid-based therapies have become the third major drug class after small molecules and antibodies. The role of nucleic acid-based therapies has been strengthened by recent regulatory approvals and tremendous clinical success. In this review, we look at the major obstacles that have hindered ...
ALS: From Genetic Complexity to Treatment Challenges and Advances
Amyotrophic Lateral Sclerosis (ALS), commonly called Lou Gehrig's disease, is a neurodegenerative disorder of the motor neurons that causes progressive muscle weakness and respiratory failure (1). ALS is the most common motor disease among adults (2), affecting around 60,000 people in the U.S. and Europe. Life expectancy for those with the fatal disease is typically 2-5 years after diagnosis ...
Personalized ASO Provides Improvements for a Girl with KAND, an Ultra-rare Disease
Within the world of rare diseases exist conditions so sparse and infrequent they’re called nano-rare ...
Angelman Syndrome Therapies Show Positive Results in Early Phase Clinical Trials
A clinical trial for a drug designed to treat the rare neurodevelopmental disorder known as ...
Encouraging Progress in the Hunt for a Huntington’s Treatment
Described as a combination of Alzheimer's disease, Amyotrophic lateral sclerosis, and Parkinson's disease all at ...
Sequence- and Structure-Dependent Cytotoxicity of Phosphorothioate and 2′-O-Methyl Modified Single-Stranded Oligonucleotides
Authors: Laura V. Croft Mark Fisher, Tabassum Khair Barbhuiya, Serene El-Kamand, Samuel Beard, Aleksandra Rajapakse, Roland ...
Nucleic Acid Therapeutics: Successes, Milestones, and Upcoming Innovation
Authors: Jillian Belgrad, Hassan H. Fakih, and Anastasia Khvorova Published Online: 3 April 2024 Abstract ...
ALS: From Genetic Complexity to Treatment Challenges and Advances
Amyotrophic Lateral Sclerosis (ALS), commonly called Lou Gehrig's disease, is a neurodegenerative disorder of the ...
