News

Parent Project Muscular Dystrophy announced that the first patient with Duchenne muscular dystrophy has been dosed with microdystrophin gene therapy.

Trulance is the only treatment thought to replicate the pH-sensitive activity of uroguanylin.

A study published this week in the Orphanet Journal of Rare Diseases confirmed the importance of genetic findings in the diagnosis of Marfan syndrome.

The rate of reward center volume loss may vary between nondepressed individuals and depressed individuals across multiple age groups.

Patients living with HIV and another comorbidity demonstrated higher ART adherence than adherence for their comorbid condition.

There were decreased reports of pain when the patients believed treatment would work, and different brain regions were activated during real versus sham treatment.

More difficult-to-get infections may be tracked better through social and risk networks.

Alex Azar was confirmed as the next secretary of the Department of Health and Human Services. Is he the right man for the job?

The FDA has released a joint warning with the FTC to 4 marketers and 11 distributors of unapproved opioid addiction therapies.

Faron Pharmaceuticals announced this morning that the U.S. FDA has approved the Investigational New Drug application for Traumakine (FP-1201-lyo) for the treatment of Acute Respiratory Distress Syndrome.

Medicare expansion was linked to a 1.8% increase in early complications and a 2.6% increase in optimal care.

The ipsogen JAK2 RGQ PCR Kit received clearance from the U.S. FDA for additional use in the diagnosis of all myeloproliferative neoplasms.

Researchers compared incoming freshman students’ medication intake to upperclassmen, finding that of both groups, only 53% took their medication.

New research shows patients who briefly stop taking antiretroviral therapy in order to participate in HIV trials don’t suffer any long-term damage.

Major mutations in the LRRK2 gene are the greatest contributor to PD. They are present in 1% to 2% of the people with PD worldwide.

On his personal website Monday, legendary musician Neil Diamond revealed that he has been diagnosed with Parkinson’s disease.

Transcranial magnetic stimulation, which was approved by the FDA for drug-resistant depression in 2009, has recently experienced a growth in clinical use.

Children whose mothers had a higher intake of sugar sweetened beverages and total fructose had a 19% greater chance of developing asthma in mid-childhood.

BERG announced this morning that the U.S. FDA has granted orphan drug designation to the company’s leading product candidate BPM31510 (ubidecarenone) for the treatment of pancreatic cancer.

This morning, Sobi, announced that the FDA has issued a Study may proceed letter for the first study in humans, thereby accepting the investigational new drug application for sobi003, and the regulatory agency granted Fast Track status to the product candidate.

Patients with hepatitis C and IR can develop a resistance to antiviral therapy, as well as fibrosis, steatosis, hepatocellular carcinoma.

Marshall Summar, M.D., director of the Children’s National Rare Disease Institute, sat down with Rare Disease Report to discuss recent news and what it means for the rare disease community.

Regeneron has announced that Dupixent, a therapy for adult atopic dermatitis (AD) that is inadequately controlled by existing medications, has been granted marketing and manufacturing authorization in Japan.

Two doses of dexamethasone resulted in similar outcomes with better adherence than five days of prednisone in children with acute asthma.

A recent study from Temple University revealed a connection between exposure to female hormones and retinal diseases in aging men.

Researchers from Stanford University have developed an HIV test that could increase testing rates, and thus treatment rates.

To kick off the new year, Alnylam Pharmaceuticals, Inc., a company dedicated to the development of RNAi therapeutics, announced the expansion of its Alnylam Act.

The U.S. FDA approved the company’s Investigational New Drug application for initiation of two Phase 3 clinical trials of dasiglucagon in treating congenital hyperinsulinism.

uniQure was granted Orphan Medicinal Product Designation from the European Medicines Agency for AMT-130, an investigational gene therapy for the treatment of Huntington’s disease.

Enzychem Lifesciences has announced that EC-18, a potential therapy for Acute Radiation Syndrome, has been granted Orphan Drug Designation by the U.S. Food and Drug Administration.