PTC Therapeutics (PTCT) shared new data being presented from the Phase 3 APHENITY trial and subsequent open-label extension study at the 2025 ...

PTC Therapeutics, Inc. (NASDAQ: PTCT) today shared new data being presented from the Phase 3 APHENITY trial and subsequent open-label extension study at the 2025 American College of Medical Genetics ...

By Gina Kolata Researchers have corrected a disease-causing gene mutation with a single infusion carrying a treatment that precisely targeted the errant gene. This was the first time a mutated ...

But in about 5% to 10% of cases, the cause is hereditary. This means that the cancer is due to a gene change, called a “mutation,” that’s passed down from a parent. Researchers have ...

A wild cat with one of the rarest coat colors ever documented has been spotted in the U.S. leaving wildlife researchers stunned and raising questions about genetic mutations and environmental factors ...

Rare disease day is observed on 28 February. Credit: nambitomo via Getty Images. US-based biotechnology company Alltrna is developing transfer RNA (tRNA)-based therapies that aim to offer a broad ...

Simulator Benchmark (2D&3D) 📈: Code for data collection, training, and evaluation using simulators from Metaworld and RLBench. Metaworld are built for the latest version of the gynasim library. You ...

Today, leukemia patients undergo genetic analysis to identify mutations and select the most appropriate treatment. However, even among patients with the same mutation, disease progression and ...

Having two copies of the MTHFR gene mutation may raise your risk for certain health conditions, though more research is needed. You may have the mutation and not find out unless you undergo ...

Mutation is the process wherein the name of the property owner is changed in the government’s land revenue records. This ensures that the land records correctly reflect the name of the existing owner ...

PKU and normal haplotypes clearly show a substantial ... too simple and needs to be improved by integrating information on mutation rates and crossing-over probabilities as soon as they become ...