Latest news with #achondroplasia
Yahoo
a day ago
- Business
- Yahoo
TransCon® hGH Boosts TransCon® CNP Results in Kids with Achondroplasia by Week 26
Ascendis Pharma A/S (NASDAQ:ASND) is among the 13 Biotech Stocks with Huge Upside Potential. It reported interim Week 26 data from its Phase 2 COACH Trial, confirming that combining TransCon hGH (lonapegsomatropin) with TransCon CNP (navepegritide) raised annualized growth velocity (AGV) and height Z-scores in children with achondroplasia. A close-up view of a hand manipulating a syringe while delivering TransCon CNP into a tumor. A mean AGV of 9.14 cm/year and a +0.53 Z-score rise were attained by treatment-naïve youngsters (N=12). Children (N=9) treated with TransCon CNP in the past achieved an AGV of 8.25 cm/year with a +0.44 Z-score improvement. The majority of side effects were modest, and safety remained consistent with monotherapies. The COACH Trial is the first to assess this combination in kids between the ages of two and eleven. While TransCon CNP is undergoing FDA priority review, TransCon hGH is approved as SKYTROFA® for juvenile growth hormone insufficiency. The treatment focuses on the FGFR3-CNP signaling imbalance that is at the heart of achondroplasia, a disorder that affects more than 250,000 people worldwide. It is anticipated that the results of week 52 and a scheduled Phase 3 trial in Q4 2025 will further clarify efficacy and long-term benefits. The biopharmaceutical business Ascendis Pharma A/S (NASDAQ:ASND) uses its TransCon technology platform to significantly improve patient outcomes. While we acknowledge the potential of ASND as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock. READ NEXT: 10 High-Growth EV Stocks to Invest In and 13 Best Car Stocks to Buy in 2025. Disclosure. None.
RNZ News
19-05-2025
- RNZ News
Man jailed in Tonga after throwing a child against a wall
Photo: 123rf A man has been jailed in Tonga after throwing a six-year-old child - who has a bone growth disorder - against a wall. Matangi Tonga reported the defendant pleaded guilty to common assault and causing serious bodily harm. Justice Tupou said the charges did not reflect what, by his own admission, the defendant did. "He punched and kicked the child until his mouth bled, strangled him by the throat until his eyes almost fell out and then threw him against the wall. Those in my view are acts of a depraved mind, especially when his victim was a six-year-old achondroplasia child that was no match for him," she said. "Meanwhile, the defendant - in an attempt to justify his actions - gave three different reasons to different people. He told [the child's mother] it was because her family were gossiping about them; he told the police he was angry with [the child's mother] and while he was trying to calm down he saw [the child's] soiled nappy leak; and lastly, he told the probation officer that he was mad and angry with [the child's mother] for playing with his feelings." The defendant submitted to court that he had conveyed his apology and regretted his actions; he took responsibility for his behaviour and said he was aware of the consequences, and he had made a vow not to be violent and is taking steps to ensure what happened is not repeated. But the judge was not convinced. "I find it unacceptable that he is more concerned about any disruptions to his studies, as a result of his offending, over the damage he may have caused to the vulnerable victim. For those reasons I doubt his remorse." The defendant was sentenced to two and a half years' imprisonment, with the final 12 months of the sentence suspended for two years with conditions. It is another chapter in an epidemic of violence in the Pacific. The United Nations warns that Pacific children face alarmingly high rates of violence and abuse , which are among the highest in the world. At the inaugural Pacific Child Wellbeing Summit earlier this year, UNICEF Pacific's representative Jonathan Veitch said boys and girls in the region face significant challenges that threaten their wellbeing. He also called for increased regional collaboration and resource-sharing to strengthen child protection systems. "It is not only the right thing to do but also the smart thing to do. The impact of child protection on a country's economic and social development is significant." The Samoa Victim Support Group (SVSG) said it has noted an increase in child victims being abused sexually and violently. "It is particularly disheartening to witness young girls, as young as 8 and 9 years old, being sheltered as victims of sexual abuse by their own relatives," it said in a statement earlier this month. "One such case involves a young girl currently under SVSG's care, who was discovered by a family sleeping in front of their home." According to SVSG, the girl had been a victim of abuse and felt too frightened to return to her abusive home environment. "The public was also devastated to hear about the death of a 13-year-old girl who was sexually abused and killed by her own relative at her own home." Unicef said up to 80 percent of children in Papua New Guinea experience physical or emotional violence and widespread neglect . Last month, alleged cases of domestic violence in Fiji led to the deaths of three people . In November 2024, a UN Women report stated the prevalence of reported physical and/or sexual intimate partner violence - taking place in the previous 12 months - was 30 percent in Melanesia.
Medscape
09-05-2025
- Health
- Medscape
Fast Five Quiz: Management of Achondroplasia
Achondroplasia is the most common form of skeletal dysplasia and is associated with numerous complications that require careful monitoring and management. This form is typically diagnosed at birth or during infancy; treatment can begin around this point to mitigate future complications, though achondroplasia usually requires lifelong management. Surgical interventions are often necessary to address specific complications, and recent advances in pharmacologic treatment have also been added to the management of this condition. Regardless, achondroplasia still impacts quality of life. Do you know how to effectively manage achondroplasia? Check your knowledge with this quick quiz.
