Rare diseases policy awaits comprehensive implementation 

The National Policy for Rare Diseases (NPRD) 2021 provides a comprehensive framework for the diagnosis and treatment of rare diseases. NPRD classifies rare diseases into three categories. The first includes conditions that can be treated with a single …

Recruiting now: study to uncover genetic cause of rare diseases

From: Garvan Institute of Medical Research Recruiting now: study to uncover genetic cause of rare diseases Sydney researchers are recruiting for a study aimed at improving diagnosis, treatment and support for an estimated two million people who live with …

More Catholic clergy in China dying due to lifestyle diseases

A Catholic priest-researcher has suggested better physical and mental care for Catholic clergy in China after data revealed a 500 percent increase in their deaths, many due to lifestyle diseases, in the past three decades. Between 1994 and March this year, …

Rhythm Drug Leads to Reduced BMI in Rare Obesity Indication

Rhythm Pharmaceuticals’ MC4R agonist Imcivree helped patients with obesity related to brain damage lose weight, teeing up the drug for a label expansion in the burgeoning obesity market, where it already has a handful of approvals. Data from the Boston- …

India needs to make drugs to combat rare diseases

Apr 7, 2025 07:46 IST First published on: Apr 7, 2025 at 07:46 IST Three decades have passed since the Supreme Court held that the right to health and medical care is a fundamental right under Article 21 of the Constitution. The Directive Principle of …

Zevra Announces Closing of Sale of Rare Pediatric Disease Priority Review Voucher for $150 Million

CELEBRATION, Fla., April 07, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announced the closing of the sale …

Defying the odds: How data and cell & gene therapy are rewriting the rare disease story

Rare diseases, affect a small percentage of the population, but their collective impact is profound. The United States defines a rare disease as one affecting fewer than 200,000 individuals, while the European Union classifies it as a condition impacting …

Govt seeks corporate help forkids with ultra-rare diseases

Why fungal diseases, fuelled by climate change, may be the next global health threat

When most people think of dangerous infections, they picture bacteria or viruses. But for infectious disease specialists like Peter Chin-Hong, one of the most insidious threats lurking in hospitals and clinics today is fungal. Chin-Hong’s case list is long …

U.S. may be reverting to a time when measles deaths were not very rare, experts warn

The United States recorded three measles deaths in the first 24 years of this century. In just over three months of 2025, it has equaled that number. The most recent patient, an 8-year-old unvaccinated and previously healthy girl in West Texas, died late …