Phase III APPOINT-PNH study of investigational oral monotherapy iptacopan met its primary endpoint; second positive Phase III topline readout for iptacopan in paroxysmal nocturnal hemoglobinuria (PNH)1
Topline APPOINT-PNH data were consistent with APPLY-PNH readout and showed a significant proportion of complement-inhibitor-naïve patients treated with iptacopan achieved clinically meaningful increases in hemoglobin levels vs. baseline without the need for blood transfusions1
PNH has a significant unmet need not addressed by anti-C5 therapies; despite treatment with anti-C5s, a large proportion of people with PNH remain anemic, fatigued and dependent on blood transfusions2-5
APPOINT-PNH data to be presented at upcoming medical meeting and included in iptacopan PNH global regulatory 2023 submissions
Addex Therapeutics (SIX and Nasdaq: ADXN), a clinical-stage pharmaceutical company pioneering allosteric modulation-based drug discovery and development, today announced that the research term of its collaboration agreement with Indivior PLC (LON: INDV) for the discovery of novel oral gamma-aminobutyric acid subtype B (GABAB) positive allosteric modulator (PAM) drug candidates for the treatment of substance use disorder has been extended until June 30, 2023. As part of the amended agreement, Indivior will pay Addex an additional USD 1 million to cover additional research activities during this extended period. Addex retains exclusive rights to develop its own independent GABAB PAM program and is advancing compounds in Charcot-Marie-Tooth type 1A neuropathy (CMT1A), chronic cough and pain.
Today, in partnership with DoorDash and Uber, Walgreens announced free delivery of Paxlovid, a COVID-19 oral antiviral therapy, directly to the doorsteps of those who need it.\
Walgreens today announced the availability of free prescription same day delivery of Paxlovid, a COVID-19 antiviral therapy, offered directly to patients’ doorsteps. (Photo: Business Wire)
With more than 8,000 Walgreens locations offering same day delivery services, available to anyone who lives within 15 miles of participating Walgreens locations, the majority of Americans have access to this program, about 92% of the population.
This initiative is aimed at increasing access to COVID-19 treatment, with a focus on reaching those in socially vulnerable or medically underserved areas at a time when COVID-19 cases are beginning to rise again across the United States. According to Walgreens COVID-19 Index data, overall positivity rates reached 36 percent this weeki. This steady rise in cases reinforces the critical need for access to life-saving treatments.
"The COVID-19 pandemic has exacerbated health disparities and emphasized the need to address long-standing barriers, including access to treatments," said Anita Patel, PharmD, vice president, pharmacy services development, Walgreens. "Our pharmacy teams will continue to play a trusted and essential role in helping to keep people protected from COVID-19, including getting people vaccinated, tested and treated as safely, equitably and effectively as possible."
Eligible patients must have a Paxlovid prescription from a healthcare provider. After the pharmacy has filled the prescription and before it gets picked up, individuals can go online to select Same Day Delivery by visitingWalgreens.com/PrescriptionDelivery, the Walgreens app or by calling their store.
Apple is reportedly ditching a controversial plan to scan users’ photos stored in iCloud for child sexual abuse material, or CSAM, amid an ongoing privacy push. \
These safety tools, announced in August 2021, were meant to flag illicit content while preserving privacy. But the plans drew widespread criticism from digital rights groups who argued that the surveillance capabilities were ripe for potential abuse.
Apple put the plans on pause a month later. Now, more than a year after its announcement, the company has no plans to move forward with the CSAM-detection tool.
The company says it is developing new features that will better balance users privacy and protect children. Such parameters will allow parents to limit their child’s contacts, restrict content and screen time and provide an app store carefully curated for kids.
Apple says the best way to prevent online exploitation of children is to interrupt it before it happens. The company pointed to new features it rolled out in December 2021 that enabled this process.
\Communication safety in messages, for instance, includes warnings when questionable photos are being sent, and expanded guidance in Siri, Spotlight, and Safari Search.
The company is working on updates to communication safety in messages to cover nudity in videos and other child safety protections. Apple says it is also working with child safety professionals to make reporting incidents to law enforcement more seamless.
The company announced Wednesday it will now offer full end-to-end encryption for nearly all the data its users store in its global cloud-based storage system, making it more difficult for hackers, spies and law enforcement agencies to access sensitive user information.
Thousands of pediatricians convened in Anaheim, Calif., in early October for the American Academy of Pediatrics’ (AAP) annual conference. The group, which boasts 67,000 members in the US and around the world describes itself as “dedicated to the health of all children.”
So some audience members were shocked when Dr. Morissa Ladinsky, an associate professor of pediatrics at the University of Alabama at Birmingham, lauded a transgender teenager for committing suicide.
In an address about “standing up for gender-affirming care,” Ladinsky eulogized Leelah Alcorn, an Ohio 17-year-old who, in Ladinsky’s words, “stepped boldly in front of a tractor trailer, ending her life,” in 2014, after leaving a suicide note that “went viral, literally around the world.”
Ladinsky’s remarks were captured on video by a horrified onlooker, Oregon pediatrician Dr. Julia Mason, who expressed outrage on Twitter that Ladinsky was “glorifying suicide,” an act she described as “unprofessional and dangerous.”
That isn’t just Mason’s opinion. Technically speaking, it is also the official stance of the AAP, whose website for parents, healthychildren.org, explicitly warns that “glorifying suicide” can have a “’contagious’ effect” and inspire others to take their own lives.
Reached for comment, Ladinsky expressed “regret” about her choice of words and said it was “never my intent” to glorify self-harm. But how did this esteemed doctor wind up telling a group of physicians that a teen had, as she put it, “boldly ended her life?”
In any large organization, some members are bound to hold fringe views. But Ladinsky, who has devoted her career in part to facilitating the gender transition of teenagers, including by challenging state laws that restrict the kinds of treatment physicians can provide to them, is hardly an outlier at the AAP. And the AAP is an organization that matters a great deal.
The AAP recommended children get the COVID-19 vaccine in order to participate in sports and other activities, even as concerns mounted about its association with myocarditis.Photo by Joseph Prezioso/AFP via Getty Images
Founded in 1930 as an offshoot of the American Medical Association, the AAP is first and foremost a standard-setting body. It outlines best practices for the nation’s pediatricians, advises policymakers on public-health issues and, for many parents, is the premier authority on raising healthy kids.
In recent years, it has also become a participant in America’s culture wars. Judges have deferred to the group’s expertise in high-stakes court cases about children with gender dysphoria, who the AAP says can start socially transitioning at “any” age. During the height of COVID, schools masked toddlers — including toddlers with speech delays — based on the guidance of the AAP. Sports leagues and after-school programs mandated the COVID vaccine after the AAP strongly recommended it, even as concerns mounted about its association with myocarditis, or inflammation of the heart muscle, in young males.
Though the organization’s guidelines are framed as the consensus position of the AAP’s members, only a handful of physicians had a role in shaping them. Instead, insiders say, the AAP is deferring to small, like-minded teams of specialists ensconced in children’s hospitals, research centers, and public-health bureaucracies, rather than seeking the insights of pediatricians who see a wide cross-section of America’s children.
They also say a longstanding left-wing bias — over two-thirds of pediatricians are registered Democrats — has accelerated, turning the organization into a more overtly political body that now pronounces on issues from climate change to immigration. As rates of gender dysphoria exploded and the COVID-19 pandemic hit, that bias seeped into the organization’s medical policy recommendations, unchecked by discussion or debate.
Dr. Marty Makary worries if parents start to distrust the AAP because of its politicization, there will be more pediatric deaths.Getty Images for HBO
In 2016, the AAP established a committee on “LGBT Health & Wellness” to support “children with variations in gender presentation.” Four of the committee’s six members — Jason Rafferty, Brittany Allen, Michelle Forcier, and Ilana Sherer — work in pediatric gender clinics that prescribe puberty blockers to patients as young as 10 and cross-sex hormones to patients as young as 14.
Those treatments are part of the broader model of “gender-affirming” care that the AAP endorsed in its 2018 policy statement, “Ensuring Comprehensive Care and Support for Transgender and Gender-Diverse Children and Adolescents.” The statement, which represents the official position of the AAP, was written by a single doctor, Rafferty, and does not appear to have been reviewed by anyone else at the organization: Rafferty “conceptualized,” “drafted,” “reviewed,” “revised” and “approved” the manuscript himself, a note at the end of the paper reads. Rafferty did not respond to a request for comment.
“There was clearly no fact-checking,” one longtime AAP member said. “The AAP thought trans was the next civil-rights crusade and got boondoggled by enthusiastic young doctors.”
The 2018 statement was an extraordinary departure from the international medical consensus. Most European countries do not encourage social or physical transition until a child’s gender dysphoria has persisted for quite some time — an approach known as “watchful waiting” — in part because the dysphoria desists on its own in the majority of cases, particularly once puberty hits.
Rafferty, however, called watchful waiting “outdated” and endorsed a “gender-affirming” paradigm, in which transitioning is on the table almost as soon as a child identifies as transgender. Some of the studies he cited to support that conclusion — including a practice guideline from the American Academy of Child and Adolescent Psychiatry — actually undercut it, arguing that, more often than not, “sex-reassignment” should be deferred until adulthood.
Though the policy statement conceded that puberty blockers may pose “long-term risks” to “bone metabolism and fertility,” it did not recommend any prerequisites for obtaining drugs. They could be given out at the earliest stages of puberty — meaning to children as young as 9 — and, Rafferty insisted, were “reversible.” Since then, the gap between the AAP and the rest of the world has only grown.
Many European countries, including Britain, Finland, Sweden, and the Netherlands, are now curtailing or entirely eliminating the use of puberty blockers in children with gender dysphoria, citing both long-term health risks and a lack of evidence that they alleviate the condition.
The AAP has nonetheless maintained its support for the drugs — which it claims have the backing of the “most prominent medical organizations worldwide” — while rejecting calls for more gatekeeping.
“The AAP says kids under 10 can’t cross the street by themselves,” one pediatrician said, referencing the group’s official recommendations on pedestrian safety, “but they can change their gender. How does that make sense?”
The contrast points to a broader tension within AAP guidance: On most kitchen-table issues, from diet to screen time to exercise, the group has long encouraged a kind of safetyism, stressing the need for parental supervision and the pitfalls of pubescent judgment. Yet on trans issues, it has done nearly the opposite, suggesting that minors are mature enough to transition without their parents’ knowledge or consent.
“A family may deny access to care that raises concerns about the youth’s welfare and safety,” Rafferty’s statement says. “In such rare situations, pediatric providers may want to familiarize themselves with relevant local consent laws and maintain their primary responsibility for the welfare of the child.”
Arkansas Attorney General Leslie Rutledge’s state passed a law similar to Alabama’s, which banned puberty blockers for transgender minors.Bloomberg via Getty Images
It’s a stark departure from the way the group talks about other forms of body modification: One AAP report recommends that “adolescents speak with their parents” before getting tattoos, because they are “permanent,” “difficult to remove” and “involve significant consequences.”
By 2019, Rafferty’s guidance was eliciting quiet concern among rank-and-file doctors affiliated with the AAP. “Normie pediatricians were like, ‘what’s going on,’ ” one doctor said, recalling the hushed conversations she had in the hallways of the AAP’s 2019 national conference, which featured a panel on gender-affirming care. Gender specialists, on the other hand, “considered themselves life-saving heroes.”
Rather than promoting dialogue or compromise between the two camps, the AAP sought to stifle dissent. In October, it urged the Department of Justice to investigate critics of “gender affirming” care, arguing they were spreading “disinformation” that puts lives at risk. That move came after the organization barred the Society for Evidence-based Gender Medicine, which advocates the watchful-waiting approach, from being an exhibitor at its national conference last year.
In August, it also blocked a resolution calling for a review of the AAP’s current guidance on puberty blockers, which the head of Boston Children’s Hospital’s gender clinic, Jeremi Carswell, says are “given out like candy” at her clinic.
The stifling of dissent has created an illusory medical consensus that nonetheless exerts extraordinary influence over public policy and debate. Courts have cited the AAP in cases about transgender children — Eknes-Tucker v. Marshall, for example, in which an Alabama District Court blocked a law banning puberty blockers, cross sex hormones and gender reassignment surgeries for transgender minors (the case is now on appeal). Talking heads, meanwhile, have invoked the AAP to shut down criticism of childhood gender transition.
In October, Jon Stewart berated Arkansas Attorney General Leslie Rutledge after her state passed a law similar to Alabama’s, arguing that she was bucking the AAP’s “peer-reviewed” guidelines. Banning puberty blockers would be as backwards as banning chemotherapy, Stewart said. He did not mention that the Swedish National Board of Health and Welfare had, in February, recommended halting hormonal gender treatment for minors except in tightly limited circumstances.
The National Institutes of Health has funded one study on the long-term effects of puberty blockers, which is being conducted by four university-affiliated gender clinics — including the one at Boston Children’s, the place that acknowledged prescribing blockers “like candy.” The study, which began in 2015, has yet to report its findings, and the authors have not declared any conflicts of interests.
Jon Stewart went after Leslie Rutledge following the passage of the law.Charles Sykes/Invision/AP
At stake in all this, said Marty Makary, a surgeon and public policy researcher at Johns Hopkins Medicine, is not just COVID lockdowns or puberty blockers but the credibility of the medical establishment itself.
“The AAP still puts out many important recommendations that parents should follow,” Makary said, citing the group’s support for the measles vaccine and its guidance on preventing sudden infant death syndrome. “If parents start to distrust the AAP because of its politicization, I worry we’ll see more pediatric deaths.”
Other doctors described families — including families in deep blue areas — who have developed a reflexive distrust of anything the AAP says.
“I now hear parents mock the AAP over even nonpolitical guidance like breastfeeding recommendations,” a pediatrician in Portland, Ore., said. “They’re just tuning everything out.”
For Vinay Prasad, a professor of epidemiology and biostatistics at the University of California, San Francisco, it’s hard to blame them.
“The reason to trust modern doctors over ancient healers is that more of what we tell you to do is justified by well-done studies,” Prasad said. “But how do we hold that perch when we just make stuff up?”
At least three partially vaccinated children in Central Ohio have contracted measles, marking the first cases in the region'soutbreakthat have not been among unvaccinated children.
Fifty nine cases had been confirmed as of Dec. 7, according to a dashboard run by the health department in Columbus. All but three of those cases were among unvaccinated children. The ages of the three partially vaccinated children who became infected are unclear, though the city's dashboard said some of the individuals may not have been eligible for their second dose of the measles-mumps-rubella shot due to their age. The CDC recommends children get their first dose of the MMR vaccine between 12 and 15 months, and the second shot between ages 4 and 6.
Three more notes on the outbreak:
1. More than half of cases have occurred among kids younger than 2. Fourteen have occurred among kids 3 to 5, and five of the children are between the ages of 6 and 17.
2. The outbreak has hospitalized 23 patients, who have been seen at Columbus-based Nationwide Children's Hospital, according to CNN. "The mainstay of treatment for all children with measles is supportive care," Matthew Washam, MD, the hospital's medical director of epidemiology and infection control, told CNN.
"In the hospital, this can include intravenous fluids, antibiotics for secondary bacterial infections. Some children with measles may also be treated with vitamin A given the association of lower vitamin A levels with more severe measles illness," he said.
3. Health officials anticipate the outbreak, which the region started investigating in early November, to last for several months. Mysheika Roberts, MD, public health commissioner at the Columbus health department said health officials are "pretty confident" the outbreak is tied to one of four separate cases reported over the summer among individuals who became infected after traveling to a country where the disease is endemic.
Sen. Ron Johnson (R-Wis.) held a panel on COVID-19 vaccines in Washington on Dec. 7, featuring experts including Dr. Robert Malone and Dr. Peter McCullough.
Experts discussed vaccine development, vaccine composition, data from insurance and adverse event systems, and other topics.
Here are four takeaways from the panel.
Sen. Ron Johnson (R-Wis.) speaks during a hearing in Washington on Jan. 24, 2022. (Drew Angerer/Getty Images)
Insurance Experts Record Jump in Excess Mortality
Edward Dowd, a former BlackRock analyst now with the Humanity Project, showed data from the Society of Actuaries 2021 Group Life Insurance survey that showed a jump in excess mortality among young and middle-aged adults starting around the time the vaccines began being administered.
The only thing that changed at the time, Dowd said, was “vaccines and mandates.”
He pointed out that Denmark and the United Kingdom, among other countries, have stopped recommending or entirely halted vaccination of young, healthy people because of growing concerns over side effects like heart inflammation that can lead to death.
“Why are our health authorities still pushing this vaccine if other countries are backing off?” Dowd asked.
Representatives from major U.S. health agencies, including the Centers for Disease Control and Prevention (CDC), were invited to the discussion but did not attend.
Josh Stirling, an insurance analyst, presented an analysis of data from the United Kingdom that concluded death rates were higher among the vaccinated as of May 2022.
Doctors Report Increase in Heart Inflammation Since Pandemic Started
Several vaccines have been linked to side effects such as myocarditis, a form of heart inflammation that can turn deadly.
Doctors testifying during the panel said they’ve seen an increase in patients with the inflammation.
“It’s been very high,” Dr. Reneta Moon, a clinical associate professor at the Washington State University College of Medicine.
Moon said she saw a handful of cases in her 20 years of practicing before the pandemic. The number has jumped since the pandemic started, she said.
Dr. Kirk Milhoan, a pediatrician based in Hawaii, said he’s also seen more cases.
Milhoan said that the researchthat’s emerged shows myocarditis and a related condition, pericarditis, are caused by the Moderna and Pfizer vaccines.
Moderna and Pfizer have not responded to requests for comment on heart inflammation.
The studies show that the spike protein, which the vaccines cause the body to make, is “cardiotoxic and cause the heart to be inflamed,” Milhoan said. “Let that sink in, the current public health plan is asking our own body to make a cardiotoxin.”
Dr. Harvey Risch, professor emeritus of epidemiology at the Yale School of Public Health, in New York on July 7, 2022. (Bao Qiu/The Epoch Times)
Young People at Little Risk From COVID-19
Dr. Harvey Risch, a professor emeritus of epidemiology at the Yale School of Public Health, presented data from the CDC that show young people face little risk from COVID-19.
The share of infection among those aged 0 to 17 that led to death, for instance, was just 0.01 percent through September 2021, while the share was 0.05 percent among those 18 to 29.
“When you have such low or nonexistent mortality in these low age groups, the potential severe adverse effects of the vaccine will surmount the nonexistent mortality of these age groups and therefore, what we’ve been told, that everybody has to be vaccinated … had no reason to be there in the first place, because there was no mortality they were trying to prevent,” Risch said.
The share was much higher among older people. Among those 85 and older, for example, the share was 24.6 percent.
Because vaccines have little to no impact on transmission or infection, the only point of getting vaccinated is for treatment, but most young people “had no reason to choose that when mortality from infection is orders of magnitude is much less than from vaccination,” Risch said.
No Strong Trials
As the vaccines have performed worse and worse against infection, health officials say they should still be taken to protect against severe disease.
But McCullough, chief scientific officer of The Wellness Company, said that no randomized, double-blind, placebo-controlled trial has shown the vaccines reduce hospitalization or death.
Such trials, known as RCTs, are generally considered the highest form of evidence for a drug.
Both the emergency authorization and approval for the vaccines have been on the basis of preventing infection. In fact sheets, prospective recipients are told the vaccines “have been shown to prevent COVID-19.” There’s no mention of severe illness or death.
