Please confirm you are human
This browser or connection looks automated. Press and continuously hold the control for 3 seconds to enable Google-hosted web results and, when separately allowed, AI-assisted answers.
A successful check enables 100 search requests. Interactive access does not authorize scraping, systematic collection, or reuse of search output.
News
Meet Professor Gina Ravenscroft: the inaugural Dr Patricia Kailis Chair in Rare Diseases
2+ hour, 46+ min ago (471+ words) Shaping the next generation of global leaders. Why genetics and rare disease research? I started my UWA journey in 2001 as an undergraduate and fell in love with research as an Honours student in the Physiology Department. Through the mentorship of…...
Spotlight On: Ultragenyx sets $2.7 million price for newly approved GSDIa gene therapy
11+ hour, 14+ min ago (15+ words) FirstWord Pharma...
Abcuro Adds $66M to Try Again in Rare Inflammatory Disorder With No Approved Drugs
12+ hour, 14+ min ago (771+ words) Abcuro will apply the capital to a study that could support a biologics license application in inclusion body myositis, a rare inflammatory disorder that leads to worsening muscle weakness. A Phase 2/3 study failed earlier this year, but Abcuro saw positive…...
Symptoms of super-rare disorder Marchiafava-Bignami explained
19+ hour, 28+ min ago (658+ words) The Five Heartbeats star's family say his death came from heart failure combined with a rare neurological condition Michael Wright, the actor best known for playing Eddie King Jr. in the 1991 cult classic musical drama The Five Heartbeats, has died…...
16-year-old boy who spent five years in a pond airlifted for treatment as doctors investigate skin condition
20+ hour, 49+ min ago (533+ words) The teenager has now been flown to Mumbai after neighbors raised concerns about his health A 16-year-old boy who has spent much of the past five years sitting in ponds and rivers is finally receiving specialist medical care after a…...
FDA nod to Ultragenyx gene therapy for rare GSDIa disorder
1+ day, 17+ hour ago (59+ words) GSDIa is caused by pathogenic variants in the G6PC gene NATCO Pharma’s Visakhapatnam facility receives 4 observations in US FDA inspection C2N Diagnostics’ PrecivityAD2 becomes first FDA-cleared Alzheimer’s blood test for adults aged 40+ SNEHA reports significant gains in child and maternal health across Mumbai’s…...
Younger Lung Cancer Patients Have Treatable Genetic Changes
1+ day, 22+ hour ago (240+ words) Aug 21, 2026 | Lung Cancer Younger adults with non-small cell lung cancer (NSCLC) are significantly more likely than older patients to have genetic alterations that can be matched with targeted therapies, according to findings to be presented at the 2026 World Conference on…...
DANDELION Computational Tool Identifies Previously Unknown Asthma-Related Genes and Pathway
2+ day, 8+ hour ago (264+ words) At first, he was skeptical. “Xuanyao showed me a list of genes, and we didn’t recognize almost any of them,” Nóbrega said. “I thought that either this is going to be really cool and groundbreaking or it’s going to be…...
Ultragenyx Receives Accelerated FDA Approval For Metabolic Gene Therapy
2+ day, 12+ hour ago (225+ words) Rare disease specialist Ultragenyx’s gene therapy Genglycos has received accelerated approval from the FDA for treatment of glycogen storage disease type Ia (GSDIa), a rare metabolic condition. This marks the fifth FDA approval for the California-based biotech, which has already…...
MRI of facial nerve may detect motor neuron damage in SMA
2+ day, 14+ hour ago (453+ words) Thinner facial nerves correlate with lower physical function Written by Margarida Maia, PhD | Aug. 21, 2026 MRI measurements of the facial nerve, which controls the muscles used for facial expression, may offer a simple way to detect damage to motor neurons — nerve…...