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
- Rare Disease News
- [email protected]
ALS Drug Facing Possible Rejection by the European Medicines Agency
- June 9, 2023
New Updates are Available on a Phase 2 Trial Evaluating CS1 for PAH
Experimental Therapy for Hemophilia Meets Phase 3 Trial Endpoints
A Newly Initiated Phase 1 Study Will Evaluate AIV007 for Wet AMD, DME
PODCAST: WAIT, HOW DO YOU SPELL THAT?
PATIENT WORTHY SERIES
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
THE LATEST FROM PATIENT WORTHY
Spinal Muscular Atrophy
Teen with SMA Uses Art to Raise Rare Disease Awareness
In March 2023, 14-year-old Rylie Erbacher visited Washington, D.C. for a one-of-a-kind experience. The teen, who has spinal muscular atrophy (SMA) type 2, was one
Osteopetrosis
SIS-101-ADO Granted Orphan Drug Designation for Autosomal Dominant Osteopetrosis Type 2
Have you ever heard of the Orphan Drug designation? This designation, created through the Orphan Drug Act and granted by the U.S. Food and Drug
Crohn's disease
Rinvoq Now Approved for Crohn’s Disease
Rinvoq (upadacitinib) is a prescription JAK inhibitor that works by blocking pro-inflammatory signaling within cells. In the past, Rinvoq was approved for indications like rheumatoid
Bile duct cancer
A Glimmer of Hope for Patients with Advanced Biliary Tract Cancer
For patients who were given a diagnosis of advanced, inoperable (unresectable) biliary tract cancer, an article appeared in Healio with some encouraging news.
Acute lymphoblastic leukemia
New Cell Therapy Approved by the FDA to Reduce Risk of Infection After Transplant
Omisirge (omidubicel) is the first allogeneic (from a donor) product for SCT that has been the recipient of FDA approval. The FDA’s nod moves
Sickle cell anemia
Study of the Week: Hydroxyurea Underused in Pediatric Sickle Cell Anemia Patients Despite Recommendations
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
Glioblastoma
Glioblastoma: Current Therapies and Recent Therapeutic Advances
Republished with permission from Personalize My Medicine, a Patient Worthy partner organization. Written by Eleanor Doherty Glossary Antibody, Antidepressant, Antigen, Apoptosis, Astrocyte, CAR-T Cell Therapy, Atypia, Clinical Trial, CNS, Dendritic Cell Vaccine, Epidermal Growth Factor, Ex-Vivo, Glioblastoma
Rare Disease
RECAP: NORD’s Living Rare, Living Stronger Patient & Family Forum
The National Organization for Rare Disorders (NORD), a Patient Worthy partner, formed in 1983 to provide support and education to people and families living with
Fabry Disease
Elfabrio Now Approved for Adults with Fabry Disease
On May 10, 2023, reports EMPR, the U.S. FDA approved Elfabrio (pegunigalsidase alfa-iwxj) for adults who are living with Fabry disease. Developed by Protalix Biotherapeutics
Fanconi Anemia
Aggies’ Head Coach Jimbo Fisher Says Gene Therapy Could Cure Son’s Fanconi Anemia
In 2014, head coach Jimbo Fisher led the Florida State Seminoles to win the BCS National Championship Game. He’s now head coach of the Texas
OF INTEREST
PATIENT STORIES
UPCOMING EVENTS
SIGN UP FOR OUR NEWSLETTER
FOLLOW US ON INSTAGRAM
