Applied Therapeutics, Inc. has taken a significant step forward in the treatment of Charcot-Marie-Tooth disease caused by Sorbitol Dehydrogenase deficiency (CMT-SORD), with the presentation of full 12-month clinical results and new topline data from 18 and 24 months at the Peripheral Nerve Society 2025 Annual Meeting. The data underscores the potential of govorestat (AT-007) to alter the course of this rare, progressive neuromuscular disease, offering hope to patients worldwide.

The findings revealed a slowing of disease progression observed via MRI at 24 months, alongside improvements in outcomes and a reduction in sorbitol levels maintained through the same period. Notably, a statistically significant correlation was found between the absolute reduction in sorbitol and changes in the 10MWRT and CMT-FOM Lower Limb domain at 12 months. Although the primary endpoint of 10MWRT at 12 months was not statistically significant, the company has since removed this measure from the CMT-FOM clinical outcomes assessment based on the ACT-CMT study results.

Further bolstering the case for govorestat, treatment in CMT-SORD-Null rats showed a highly statistically significant 37% reduction of sorbitol in the sciatic nerve, mirroring the peripheral neuropathy experienced by patients. The drug has been generally safe and well tolerated, with the company remaining on track for a potential NDA submission in 2025. This commitment reflects Applied Therapeutics' dedication to addressing unmet needs in rare diseases.

The positive data has not gone unnoticed by the investment community, with Applied Therapeutics' shares experiencing a notable surge. Retail traders, in particular, have shown heightened interest, as evidenced by a 629% surge in 24-hour message volume on Stocktwits. The optimism among investors underscores the potential market impact of govorestat, should it receive regulatory approval, marking a pivotal moment for both the company and patients affected by CMT-SORD.

Sandy Gall, the revered war correspondent and long-standing ITN broadcaster, has died at the age of 97. Known for his fearless reporting from some of the most dangerous conflict zones around the world, Gall's career spanned over six decades, during which he became one of the most trusted voices in British journalism. His passing marks the end of an era for war reporting and broadcast journalism.

Gall's journey in journalism began in the early 1950s with Reuters, covering significant global events such as the Suez Crisis and the assassination of President Kennedy. His transition to ITN in 1963 marked the start of a legendary tenure that would see him cover pivotal moments in history, including the Vietnam War and the Soviet-Afghan War. Gall's dedication to reporting from the front lines, often without official clearance, underscored his commitment to bringing the truth to the public.

Beyond his achievements in journalism, Gall was also known for his humanitarian efforts. After retiring from ITN in 1992, he founded Sandy Gall's Afghanistan Appeal, a charity aimed at aiding victims of war, particularly those injured by landmines and children in refugee camps. His work off the field was a testament to his compassion and dedication to making a difference in the lives of those affected by conflict.

Sandy Gall's legacy is not only defined by his groundbreaking reporting but also by his mentorship to younger journalists and his unwavering integrity. As tributes pour in from colleagues and admirers worldwide, it's clear that Gall's contributions to journalism and humanitarian efforts will continue to inspire future generations. His was indeed a great life, generously and courageously lived.