ALS Drug Facing Possible Rejection by the European Medicines Agency

Rose Duesterwald - June 9, 2023

pulmonary arterial hypertension

New Updates are Available on a Phase 2 Trial Evaluating CS1 for PAH

HemophiliaHemophilia AHemophilia B

Experimental Therapy for Hemophilia Meets Phase 3 Trial Endpoints

Diabetic macular edemaWet macular degeneration

A Newly Initiated Phase 1 Study Will Evaluate AIV007 for Wet AMD, DME

PODCAST: WAIT, HOW DO YOU SPELL THAT?

PATIENT WORTHY SERIES

Rare Classroom: Stevens-Johnson Syndrome

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

World Orphan Drug Congress USA 2023: The Forefront of Rare Disease Drug Development

The World Orphan Drug Congress USA 2023 was held from May 23-25, 2023 at the Gaylord National Resort & Convention Center in National Harbor, MD.

Rare Community Profiles: How the Seena Magowitz Foundation is Working to Change the World for People Living with Pancreatic Cancer

Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease

Study of the Week: RNA Sequencing Platform Could Improve Pediatric Rare Disease Diagnosis

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

Diffuse Large B-cell Lymphoma

The FDA Approves Roche’s Antibody Drug Polivy to Treat Lymphoma Patients

In March the FDA’s expert advisers voted 11 to 2 in favor of approving Roche’s antibody drug Polivy to treat lymphoma patients. According to an

Nonalcoholic fatty liver disease

Bariatric Surgery Could Improve Outcomes for Obese Individuals with NAFLD

Bariatric surgery refers to surgical procedures that can help people with obesity to lose weight when other means have not worked. This type of surgery

Rare Disease

ABBV CLS 7262 is Being Evaluated as a Potential Treatment for Vanishing White Matter Disease in the Brain

The safety, pharmacokinetics (absorption), and tolerability of the investigational eIF2B activator ABBV-CLS-7262 is being evaluated in a Phase 1b clinical study to treat patients with

Insurance

Indiana Legislature Passes Bill Protecting Patients from Paying More Than Their Insurers

PhRMA Public Affairs recently carried the good news that Governor Holcomb of Indiana has joined the governors of Arkansas and West Virginia by signing a

Multiple Sclerosis

Immunic Inc. Announces Promising Pre-Clinical Findings for Multiple Sclerosis

This week PR Newswire carried an announcement by the biotechnology company Immunic Inc. of preclinical data confirming that Vidofludimus Calcium (IMU 838) may be a

Batten Disease

Boy with Batten Disease Pens Book About Marshmallow, His Cane

7-year-old Peter Heath loves to tell jokes, play with Legos, hang out with his friends, and spend time outside. Guided by his white cane, which

SHINE Syndrome

Mom Runs in Flying Pig Marathon to Raise SHINE Syndrome Awareness

Last year, 35-year-old Jenna Neduchal ran her first Flying Pig Marathon. This annual event serves to unite the community, create an accessible event for all,

Hereditary Spastic Paraparesis

Organoids Could Be Used to Find Effective HSP Treatments

There are no cures or treatments available for people with hereditary spastic paraplegia (HSP) type 56, or SPG56. While the current standards-of-care focus on symptom

Alexander disease

ICYMI: Two Professors Receive $74K Grant to Study Alexander Disease

First described in medical literature in 1949, Alexander disease is an extremely rare neurological disorder and form of leukodystrophy. Despite nearly 74 years having passed