Expanded-access use of elamipretide in a critically ill patient with Barth syndrome

Endothelins (ETs) are potent vasoactive peptides critical in the human body's various physiological and pathological processes. One of the most promising therapeutic strategies for treating pulmonary arterial hypertension (PAH) involves counteracting the effects of these endothelins using a class of drugs known as endothelin receptor antagonists.
ETs are synthesized through a complex sequence of enzymatic steps, primarily involving an enzyme referred to as endothelin-converting enzyme...

Individuals with Barrett's esophagus are often asymptomatic, but they may experience symptoms commonly associated with GERD, such as heartburn and acid regurgitation. Additional symptoms can include difficulty swallowing, chest pain, unintentional weight loss, blood in the stool (which may appear black, tarry, or bloody), and episodes of vomiting.
To diagnose Barrett's esophagus, healthcare providers often recommend an endoscopy for those showing symptoms of acid reflux. The procedure...

Diarrhea-predominant irritable bowel syndrome (IBS-D) is a subtype of IBS characterized primarily by frequent, loose, or watery stools, abdominal pain, and abdominal discomfort. Therapeutic approaches to managing IBS-D include dietary changes, stress management techniques, and pharmaceutical interventions.
Two specific drugs used in the treatment are alosetron (Lotronex) and eluxadoline (Viberzi). Alosetron, a 5-HT3 antagonist, works by slowing the movement of stools in the gut, reducing bowel...

Endoscopic Retrograde Cholangiopancreatography (ERCP) is a diagnostic procedure that combines endoscopy and fluoroscopy to diagnose and treat conditions related to the bile ducts, pancreatic ducts, and gallbladder. This procedure is beneficial for identifying and addressing blockages, gallstones, strictures, and tumors within the biliary or pancreatic systems. ERCP is both diagnostic and therapeutic, offering the ability to visualize and treat identified problems in one session.
Patient...

A Barium Enema, or a lower GI series, is a specialized radiographic examination designed to visualize the lower gastrointestinal tract, specifically the colon and rectum. This procedure is instrumental in diagnosing various conditions such as colorectal cancer, polyps, diverticulosis, and inflammatory bowel disease.
Procedure Details
The examination begins by inserting a lubricated rectal tube into the patient's rectum to administer a radiopaque barium solution. The barium flow is carefully...

Enteral nutrition encompasses various methods of delivering nutrition directly to the gastrointestinal (GI) tract, bypassing traditional oral intake. It is particularly beneficial for patients who cannot eat by mouth but have a functioning digestive system. Key methods include nasointestinal feeding, gastrostomy, and jejunostomy, each suited to different clinical scenarios based on the patient's needs and condition.
Nasointestinal Feeding
Nasointestinal feeding involves placing a tube...