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PATIENT WORTHY SERIES

Study of the Week: Some Genetic Tests Miss Familial Hypercholesterolemia

Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we

Rare Classroom: Familial Partial Lipodystrophy

Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on

Compassion Corner: Should Care be Patient Centric? Does Compassion Matter?

684 words, 9% matched vs 1,329 words, 4% matched Compassion [kuhm–pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by

Rare Classroom: Diabetic Retinopathy

Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on

Rare Disease

Two Events In This Doctor’s Life Convinced Him To Add Compassion On His List of Medical Interventions

Dr. Duneel Dhand, an internal medicine physician and author, told KevinMD about two events in his life that motivated him to treat his patients with

Congenital Athymia

Novel Investigative Therapy for Pediatric Congenital Athymia Submitted to FDA for Approval

Enzyvant has just announced that they have resubmitted their application of RVT-802 to the FDA as a therapy for pediatric congenital athymia. Their first application

Rare Disease

Data Sharing Allows for More Rare Disease Diagnoses

Diagnosis can be difficult to achieve for rare disease patients. Some patients experience years of searching for the correct diagnosis, with others receiving the wrong

Adult growth hormone deficiency

Rare Classroom: Adult Growth Hormone Deficiency

Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on

Amyotrophic Lateral Sclerosis

New Pediatric Form of ALS Discovered

Normally, amyotrophic lateral sclerosis (ALS), a progressive neurodegenerative disorder, affects individuals between ages 50-60, with prevalence increasing with age. Once diagnosed, patients typically survive

Lennox-Gastaut syndrome

6th Annual LGS Foundation Walk ‘n’ Wheel to Take Place June 5

According to a recent news release, the Lennox-Gastaut Syndrome (LGS) Foundation shared that its 6th Annual Walk ‘n’ Wheel event will take place on

Progressive multifocal leukoencephalopathy

First Patient Dosed in NT-I7 Trial for PML

On May 24, 2021, biopharmaceutical company NeoImmuneTech, Inc. shared that the first patient was dosed in a pilot study centered around NT-I7 (efineptakin alfa).

Rare Disease

African Americans Are at a Higher Risk of Transthyretin Amyloid Cardiomyopathy

Pfizer Rare Disease is an approach taken by pharmaceutical giant Pfizer to develop treatments for rare and orphan diseases. In the past, the company has

Limb-girdle muscular dystrophy

FDA Clears LION-101 IND for LGMD21/R9

In a press release from late May 2021, AAV gene therapy company and Bayer AG subsidiary Asklepios BioPharmaceutical, Inc. (“AskBio”) shared that the FDA approved

Anaplastic Lymphoma Kinase-Positive Non-Small Cell Lung Cancer

Real-World Data on Brigatinib as a Treatment for ALK+ Non-Small Cell Lung Cancer

Brigatinib has been approved for the treatment of ALK+ metastatic non-small cell lung cancer (NSCLC), as well as ALK+ NSCLC patients who have received prior