Reports of seven deaths in patients who had taken Neurocrine Biosciences’ Vykat and the official termination of Aardvark Therapeutics’ Phase 3 trial after cardiac complications marked a tough day for the Prader-Willi syndrome community.

Original title: “Deaths reported after treatment with Neurocrine’s approved Prader-Willi drug”

Rhythm Pharmaceuticals’ Imcivree reduced fat—while boosting muscle—in patients with Prader-Willi syndrome.

Original title: “Rhythm’s obesity drug scores ‘better than expected’ weight loss in rare genetic disease”

review of estimates of birth incidence and population prevalence over time and between countries of the rare neurodevelopmental condition prader willi syndrome

Original title: “Review of estimates of birth incidence and population prevalence over time and between countries of the rare neurodevelopmental condition Prader-Willi syndrome.”

sleeve gastrectomy in paediatric and transition age patients with prader willi syndrome a 5 year longitudinal study

Original title: “Sleeve Gastrectomy in Paediatric and Transition Age Patients with Prader-Willi Syndrome: A 5-Year Longitudinal Study.”

The company plans to unblind the late-stage study that was halted due to safety concerns, which could lead to an overhaul of its entire development program.

Original title: “FDA hold puts Aardvark Prader-Willi drug in limbo”

Aardvark Therapeutics had previously voluntarily suspended studies of ARD-101—and a related asset called ARD-201—after detecting anomalous echocardiographic readings in healthy volunteers that could indicate reduced heart efficiency.

Original title: “Future of Aardvark’s Prader-Willi drug in doubt as FDA slaps full hold on program”

metabolic profile in prader willi syndrome patients followed at a single expert center of rare endocrine diseases

Original title: “Metabolic profile in Prader-Willi syndrome patients followed at a single expert center of rare endocrine diseases.”

The deal hands Neurocrine a “controversial” medicine, Vykat, that achieved $190 million in sales last year but faces persistent questions about its growth trajectory and safety, according to Wall Street analysts.

Original title: “Neurocrine to acquire Prader-Will drug in $2.9B Soleno buyout”

While an acquisition is a good exit for Soleno Therapeutics, the company’s acceptance of Neurocrine Biosciences’ $53-per-share offer came as a surprise to Stifel analysts given the potential growth of Vykat XR, approved last year for extreme hunger in patients with Prader-Willi syndrome.

Original title: “Neurocrine Nabs Rare Obesity Disorder Drug in ‘Surprising’ $2.9B Soleno Deal”

pharmacological management of obesity in prader willi syndrome what does the future hold

Original title: “The pharmacological management of obesity in Prader-Willi syndrome: what does the future hold?”

Aardvark Therapeutics is down 54% since Friday after the biotech said it detected “reversible cardiac observations” in a healthy volunteer study of its drug to treat extreme hunger in patients with the rare genetic disease.

Original title: “Aardvark Tanks After Pausing Prader-Willi Study on ‘Surprise’ Safety Signals”

The detection of certain cardiac issues in a study of healthy volunteers led Aardvark to pause a Phase 3 trial only months before an expected data readout.

Original title: “Safety concerns spur Aardvark to halt key Prader-Willi drug trial”

Aardvark Therapeutics is voluntarily pausing its late-stage trial testing its experimental treatment for a type of rare genetic disease after it found heart-related side effects in healthy volunteers, the company said on Friday. Feb 27 (Reuters) - Aardvark Therapeutics (AARD.O), opens new tab is voluntarily pausing its late-stage trial testing its experimental treatment for a type of rare genetic disease after it found heart-related side effects in healthy volunteers, the company said on Friday. Aardvark was testing ARD-101 as a potential treatment for hyperphagia, or feelings of intense and persistent hunger, in patients with a rare genetic disorder called Prader-Willi syndrome. The company said it is conducting a comprehensive review of the data to inform next steps. "We are committed to advancing the ARD-101 clinical program and we are evaluating optimal therapeutic dosing levels to support its progress," CEO Tien Lee said in a statement. Currently, only one FDA-approved treatment exists for hyperphagia, a once-daily oral pill called Vykat XR by Soleno Therapeutics (SLNO.O), opens new tab. Last year, Acadia Pharmaceuticals (ACAD.O), opens new tab said it will discontinue development of its intranasal therapy to treat Prader-Willi syndrome after it failed to show a benefit in patients in a late-stage trial.

Original title: “Aardvark pauses late-stage trial testing rare disease treatment | Reuters”

understanding the burden of endocrine and metabolic disorders in prader willi syndrome data from the italian registry

Original title: “Understanding the burden of endocrine and metabolic disorders in Prader-Willi syndrome: data from the Italian registry.”

oxytocin in infants with prader willi syndrome to improve dysphagia and disease trajectory

Original title: “Oxytocin in infants with Prader-Willi syndrome to improve dysphagia and disease trajectory.”

Rare Disease Day is a global movement to raise awareness and advocate for the more than 300 million people worldwide living with a rare disease. Held annually on the last day of February, this day unites individuals, Over the years, as a special-needs parent navigating the Special Education system for Sophie, Jennifer became interested in learning as much as possible about that process. She began by attending PWSA (USA)’s first Wyatt Special Education Advocacy Training in March of 2013, and went on to complete a rigorous 9-month Special Education Advocacy Training course with the Council of Parent Advocates and Attorneys in May of 2015. She obtained her PhD from Capella University in Education focusing her research on Prader-Willi Syndrome. Dr. McTighe is also a graduate of the William & Mary Law School’s Institute for Special Education Advocacy. Dr. McTighe is currently the chair of the Special Education Advisory Board for the Prader-Willi Syndrome Association-(USA). In addition to Evan’s work with parents, he helped create PWSA (USA)’s Wyatt Special Education Advocacy Training (WSEAT) in 2013, the PWSA (USA) Special Education Advisory Board (SEAB) and created PWSA (USA)’s e-letter “School Times”. Evan’s special education advocacy training includes graduating from the William and Mary College of Law’s Special Education Advocacy Institute and regularly attending the annual Special Education Law Symposium at Lehigh University. It partners with the Southern Poverty Law Center, Protection and Advocacy (P&A) programs, Legal Services Corporations (LSC) and disability organizations on major, systemic disability rights issues involving the Individuals with Disabilities Education Act (IDEA), Americans with Disabilities Act (ADA), and the federal Medicaid Act.

Original title: “Rare Disease Day 2026 - Prader-Willi Syndrome Association | USA”