Rare

Taliban forces fire on crowds in Herat, who were protesting at manhandling of women arrested over hijab dress codeA Taliban crackdown on women’s dress code in Afghanistan has escalated into a rare mass street protest in the western province of Herat, with at least two people killed by security forces.Officials made a wave of arrests in recent days targeting women and young girls accused of “improper hijab”. Residents say many families had received no information about the whereabouts or condition of those detained. Continue reading...

• Children’s Tumor Foundation (CTF) CEO Annette Bakker reports that over 5,000 potential treatments for rare diseases are currently shelved by pharmaceutical companies. • The organization is working to rescue these dormant drug candidates to provide options for patients with rare conditions who lack viable therapies. • This initiative addresses a critical gap in drug development where promising compounds are abandoned due to market size or corporate priorities.

Nauran government says its citizens are ‘friendly’ after MP spoke of serious threats allegedly made against former Australian detaineesGet our breaking news email, free app or daily news podcastThe Nauruan government has issued a rare statement insisting it is a “friendly” and “welcoming” country after a whistleblower alleged “serious threats of physical violence” were made against a group of non-citizens removed there by the Albanese government.The unexpected defence, sent shortly after midday Thursday, was mounted hours after independent MP Andrew Wilkie used his three-minute constituency statement to read claims from an anonymous whistleblower familiar with the arrangements of the secretive $2.5bn deal between Australia and Nauru. Continue reading...

While president’s last-minute endorsement of Randy Feenstra failed, he has enjoyed successes in Texas, Indiana and KentuckyZach Lahn’s victory in Iowa’s gubernatorial primary on Tuesday is a rare instance of Republican voters rejecting Donald Trump, who has used his endorsement to elevate proteges and oust rivals nationwide ahead of the November midterm elections.In the race to replace Kim Reynolds, Iowa’s Republican governor, who is not seeking re-election, the president had given Randy Feenstra, a congressman, his “Complete and Total Endorsement”, which would normally be enough to see him to victory. Instead, Lahn, a farmer and businessman, won Tuesday’s Republican primary with 38% of the vote to Feenstra’s 37.2%, according to the Associated Press. Continue reading...

• Following the resignation of Commissioner Marty Makary, the FDA is seeking a new leader to oversee the agency's commitment to accelerating treatment access for rare disease patients. • The urgency is highlighted by UX111 gene therapy, which clinical trials show can reduce toxin buildup for over eight years and slow cognitive decline if administered early. • This transition is critical because children unable to access UX111 through clinical trials continue to suffer irreversible losses while awaiting formal FDA approval.

Tragedy in Washington and near-miss in California cast in sharp relief the risk of chemical spills and explosionsFor several tense days last week, tens of thousands of southern California residents were left wondering whether a 7,000-gallon chemical storage tank would either explode or spill out into the streets.The episode cast in sharp relief the risk of chemical spills and explosions that lurk behind every corner of modern life. The methyl methacrylate that recently left the city of Garden Grove teetering on the edge of disaster is just one of many toxic chemicals commonly found in American cities. Continue reading...

• Representative Esposito is urging the FDA to accelerate the approval process for rare disease treatments, noting that 30 million Americans suffer from over 10,000 different rare conditions. • The push highlights UX111 gene therapy, which clinical trials show can reduce toxin buildup for over eight years and slow cognitive decline when administered early. • The situation is critical because standard regulatory timelines are too slow for fast-progressing diseases, leaving children to suffer permanent losses while waiting for access.