Study of the Week: Protein from Sweat is Protective Against Lyme Disease
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 Disease News
ATTR Amyloidosis: 1 in 4 Patients Experience Neurological and Cardiac Symptoms
- April 16, 2024
Danicopan Approved as Add-On Therapy for PNH Treatment
Raising Vanishing White Matter Disease (VWM) Awareness: How Ella’s Pitch Catalyzed Change
Study of the Week: Protein from Sweat is Protective Against Lyme Disease
PODCAST: WAIT, HOW DO YOU SPELL THAT?
PATIENT WORTHY SERIES
Rare Community Profiles: How Beth and Madison Advocated for Public Funding for the First Cystic Fibrosis (CF) Modulator in Canada
Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their
Rare Community Profiles: The Urgent Need for a Cure for Niemann-Pick Type C: Two Parents Share Their Perspectives
Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their
Rare Community Profiles: Kristi’s Fight to Combat Misconceptions about Prader-Willi Syndrome (PWS): “Our Children Have PWS; They Are Not PWS”
Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their
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Keratoconus
Intrastromal Lamellar Keratoplasty with Collagen Cross-Linking Improves Progressive Keratoconus
The European Society of Cataract & Refractive Surgeons (ESCRS) held its ESCRS Winter Meeting from February 15-18, 2024. During the meeting, Dr. M.S. Swathi, MD,
Niemann-Pick Type C Disease
Rare Community Profiles: The Urgent Need for a Cure for Niemann-Pick Type C: Two Parents Share Their Perspectives
Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their
Lyme Disease
This Protein from Sweat Could Offer Protection from Lyme Disease
According to reporting in Science Daily, a team of researchers affiliated with MIT and the University of Helsinki have identified a protein found in human
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April is Sjögren’s Syndrome Awareness Month
The month of April is recognized as Sjögren’s Awareness Month, a time for spreading awareness about the autoimmune disease Sjögren’s syndrome in the medical community
Prader-Willi Syndrome
Rare Community Profiles: Kristi’s Fight to Combat Misconceptions about Prader-Willi Syndrome (PWS): “Our Children Have PWS; They Are Not PWS”
Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their
Rare Disease
Study of the Week: Researchers Use Whole Human Liver for Gene Therapy Testing
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
Sarcoidosis
Cyclist Shares Her Story During Sarcoidosis Awareness Month
April is Sarcoidosis Awareness Month. During this month, people with sarcoidosis, caregivers, physicians, and other supporters come together to amplify the stories of those affected,
Myasthenia Gravis
Enspryng Falls Short in Generalized Myasthenia Gravis Clinical Study
While there is no cure for myasthenia gravis, this disorder can be managed with treatment. Current treatment options include a thymectomy, C5 protein inhibitors, and
Metabolic dysfunction-associated steatohepatitis
Updated Study Data Suggests Efruxifermin Benefits Stage 2 or 3 MASH
Over the past few years, Akero Therapeutics has experienced high highs and low lows in its efforts to advance efruxifermin, an investigational drug for people
Rare Disease
Highlights of ACMG 2024
Perhaps 70- 85% of rare diseases have a root in genetics, – which is why continuous research and reporting is so critical for those with
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