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Researchers employ novel algorithm to identify candidate cancer drugs for pulmonary hypertension

Researchers employ novel algorithm to identify candidate cancer drugs for pulmonary hypertension

Researchers illustrate the importance and value of sharing negative research results

Researchers illustrate the importance and value of sharing negative research results

Three new genetic variants linked to fibromuscular dysplasia

Three new genetic variants linked to fibromuscular dysplasia

AI algorithm accurately detects disease-causing variants in infants with rare diseases

AI algorithm accurately detects disease-causing variants in infants with rare diseases

Scientists identify gene responsible for food-dependent form of Cushing's Syndrome

Scientists identify gene responsible for food-dependent form of Cushing's Syndrome

New study aims to find early drivers of pulmonary fibrosis in at-risk patient population

New study aims to find early drivers of pulmonary fibrosis in at-risk patient population

Researchers identify mechanism that can lead to deafness in rare Norrie disease

Researchers identify mechanism that can lead to deafness in rare Norrie disease

Kawasaki Disease presents a growing threat to UK children’s heart health

Kawasaki Disease presents a growing threat to UK children’s heart health

Primary sclerosing cholangitis: Therapy with synthetic bile acid acts directly on the immune system

Primary sclerosing cholangitis: Therapy with synthetic bile acid acts directly on the immune system

New RDCA-DAP program launches to accelerate rare disease treatment innovation

New RDCA-DAP program launches to accelerate rare disease treatment innovation

Albert Einstein College of Medicine receives $5 million NIH grant for IDD research

Albert Einstein College of Medicine receives $5 million NIH grant for IDD research

First rat model recapitulates all disabling alterations experienced by patients with Morquio A disease

First rat model recapitulates all disabling alterations experienced by patients with Morquio A disease

Activated mast cell disorders potentially linked to SARS-CoV-2 immunity

Activated mast cell disorders potentially linked to SARS-CoV-2 immunity

Researchers develop a machine learning-based screening tool for genetic syndromes in children

Researchers develop a machine learning-based screening tool for genetic syndromes in children

FDA approves an intravenous medication to treat patients with Pompe disease

FDA approves an intravenous medication to treat patients with Pompe disease

Graduate student receives NIH grant to study specific molecule’s role in healthy neurodevelopment

Graduate student receives NIH grant to study specific molecule’s role in healthy neurodevelopment

Drug combination found to be safe and effective in malignant peritoneal mesothelioma patients

Drug combination found to be safe and effective in malignant peritoneal mesothelioma patients

Dying patients with rare diseases struggle to get experimental therapies

Dying patients with rare diseases struggle to get experimental therapies

Why we may never know whether the $56,000-a-year Alzheimer’s drug actually works

Why we may never know whether the $56,000-a-year Alzheimer’s drug actually works

Study uncovers mutations in Polycomb group gene as underlying cause of novel neurological disorder

Study uncovers mutations in Polycomb group gene as underlying cause of novel neurological disorder