News

World-renowned Cambridge University physicist and best-selling author Stephen Hawking died at his home in England early this morning. He was 76 years old.

Jupiter Orphan Therapeutics has submitted the first of the several highly anticipated investigational new drug applications for potential therapies that utilize Jotrol, its platform product.

Psychological stress-induced alterations of the neuroendocrine-immune system in allergic airway inflammation suggest a novel phenotype in asthma.

Development of interventions that provide strategies for young adults with ADHD to respond to influence may provide the best solution to nonmedical prescription stimulant use on campuses.

To date, there has been no assessment of CMV reactivation and CV risk in liver transplant patients.

The device will offer real-time tracking and alerts for glucose level high and low events, as well as telemedicine capabilities.

Teva Pharmaceuticals, the HDSA, and the ALS Association are partnering for the Teva CNS Target Identification Challenge, a crowdsourcing effort to seek novel targets with therapeutic potential.

New research indicates overweight patients may have worse asthma control outcomes after a year of subcutaneous immunotherapy than their normal weight counterparts.

The Medical College of Wisconsin has received clearance from the U.S. FDA for the previously unannounced IND application for the Phase 1 trial of a combination therapy for relapsed or refractory AML.

This morning, Alynylam Pharmaceuticals, Inc. has announced the Bridge the Gap initiative in which it will partner with a family affected by hATTR amyloidosis for a new book. The initiative, called , will allow the family that has lived with the rare disease for generations to share its personal accounts. The book is titled “Living a Rare Life,” and can be found, along with other educational content, at hATTRBridge.com.

Interim clinical data from the ongoing BP-004 trial suggest that BPX-501 reduces cancer recurrence rates in pediatric patients with acute myeloid leukemia and primary immunodeficiencies.

The model shows which bacteria strains will have the best chance of growth in FMT recipients.

While some studies have shown that steroids helped resolve sore throats faster, others have indicated that steroids show no effectiveness for sinusitis or bronchitis.

A joint statement from the Endocrine Society, the ADA, the AACE, and the AADE expressed strong disagreement with the ACP’s new guidelines, which raised goal A1C levels to between 7% and 8% for patients with T2D.

Anaya Mitchell was diagnosed with myasthenia gravis (MG) in the Summer of 2013. She was 13 years old. In this video, describes the challenging road she had on her way to diagnosis.

A February 2018 Type C meeting between Sarepta Therapeutics, Inc. and the Division of Neurology Products of the U.S. FDA was held to solicit the Division’s guidance on the development pathway for Sarepta’s golodirsen (SRP-4053).

On the heels of announcing that the Odyssey Outcomes trial met its primary endpoint, Regeneron and Sanofi are ensuring more affordable and timely access to patients who need alirocumab (Pralutent Injection).

Results from a global market research survey on phenylketonuria were presented, and concluded that patients’ adherence to currently prescribed supplements is a significant issue.

Treatment of HCV was associated with lower risk for other maladies in the largest western study of the relationship between antiviral treatment and extrahepatic conditions.

For treating AMD, the 3 anti-VEGF agents report similar efficacy and safety results in trials but different molecular structure and biochemical properties.

It was recently revealed that a newly-discovered biochemical pathway could assist scientists develop new ways to protect cells against the oxidative stress that is commonly associated with Huntington’s disease.

Paul Melmeyer, Director of Federal Policy for the National Organization for Rare Disorders (NORD) wants to do his part to ensure that the proper choices are being made on behalf of people affected by rare diseases.

Some of the non-modifiable risk factors found were determined to be patient-related — such as older age, female sex, and white or African descent.

A historic meeting will be held on April 6 when the FDA will host a joint Externally-Led Patient-Focused Drug Development (EL-PFDD) meeting with Debra of America and the Pachyonychia Congenita Project.

Virtually all deaths from RSV occur in developing countries, and long-term airway morbidity should be studied in those parts of the world.

Researchers isolated the neurotoxic complex crotoxin from the venom of Crotalus durissus terrificus, commonly known as the South American rattlesnake.

The therapies come on the tail of Amgen's failed attempt to receive a 6-month exclusivity extension for Sensipar.

On March 5, at the New Jersey Rare Disease Day event, hosted by the New Jersey Rare Disease Alliance, John Maslowski, CEO of Fibrocell Science, sat down with Rare Disease Report to discuss clinical development.

Data presented at the WORLDSymposium meeting in San Diego last month and published in the clinical journal Molecular Genetics and Metabolism concludes that pregnant women receiving enzyme replacement therapy (ERT) for Gaucher disease can continue taking it.

The fall of 2017 brought the approval of new gene therapies, and could usher in a new area of personalized medicine.