Kaitlynn Ely

NBC News chief foreign correspondent Richard Engel's most demanding role has taken place off-camera. He and his wife, Mary Forrest, support each other in raising their 2-year old son, Henry, a patient with Rett syndrome.

PTC Therapeutics has announced that Part 1 of the FIREFISH study has shown that RG7916, a Type 1 spinal muscular atrophy (SMA) drug, to be safe and well-tolerated in infants and babies with Type 1 SMA.

The Canadian Agency for Drugs and Technologies in Health organization’s Common Drug Review protocol often leaves rare disease patients in British Columbia without governmental payment coverage for their expensive treatments.

As the Myanmar military’s genocidal actions continue to displace Rohingya refugees, the persecuted minority group is experiencing the largest diphtheria outbreak since the 1920s.

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.

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.

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.

An orphan drug designation request for Vitality Biopharma’s prodrug VITA-100, a cannabinoid that treats pediatric ulcerative colitis, has been sent to the U.S. FDA.

Emmaus Life Sciences has announced that Endari, a medication developed to reduce acute complications in patients with sickle cell disease, is now available by prescription in the United States.

Researchers at St. Jude Children’s Research Hospital have developed a new gene therapy that creates fully-functioning immune systems in babies diagnosed with severe combined immunodeficiency, commonly referred to as the “Bubble Boy” disease.

HCell announced today that HC017AA, a treatment for Alopecia Areata in pediatric patients, has been granted Orphan Drug Designation by the U.S. FDA.

After discovering the fate of a former teammate’s child, wrestlers at Wells High School in Maine are using their sport as an incentive to raise money for Sanfilippo Syndrome.

On Monday, Shire announced that it plans to reorganize its business structure and create two distinct operational divisions; one in rare disease and another in neuroscience.

Acceleron Pharma has announced that Part 1 of the Phase 2 clinical trial of ACE-083, a drug used to increase muscle volume in facioscapulohumeral dystrophy (FSHD), has exhibited positive results.

Stephen Hawking, the notable British physicist and author of A Brief History of Time, celebrates his 76th birthday today, making him one of the longest surviving patients with amyotrophic lateral sclerosis (ALS).