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Researchers Find Immunotherapy Drug May Treat Pheochromocytoma

Researchers Find Immunotherapy Drug May Treat Pheochromocytoma

Those diagnosed with paraganglioma-pheochromocytoma, squamous cell carcinoma of the skin (cSCC), adrenocortical carcinoma (ACC), and carcinoma of unknown primary (CUP) may soon have an additional option for treatment. According to…

Continue Reading Researchers Find Immunotherapy Drug May Treat Pheochromocytoma
Faulty Proteins and Failed Genes: Understanding the Biology Behind Congenital Diseases

Faulty Proteins and Failed Genes: Understanding the Biology Behind Congenital Diseases

Patients with congenital diseases often experience a range of uncomfortable symptoms. On one side of the world, a family with erythromelalgia, a painful condition which can cause redness and swelling…

Continue Reading Faulty Proteins and Failed Genes: Understanding the Biology Behind Congenital Diseases
Effectiveness of Prenatal Surgery for Spina Bifida Provides More Hope for Babies with Myelomeningocele

Effectiveness of Prenatal Surgery for Spina Bifida Provides More Hope for Babies with Myelomeningocele

By Natalie Homan from In The Cloud Copy Spina bifida is a condition that appears when a developing baby’s neural tube doesn’t close completely. The most severe form of spina…

Continue Reading Effectiveness of Prenatal Surgery for Spina Bifida Provides More Hope for Babies with Myelomeningocele
Baricitinib Granted Breakthrough Therapy Designation for the Treatment of Alopecia Areata

Baricitinib Granted Breakthrough Therapy Designation for the Treatment of Alopecia Areata

There are currently no FDA approved treatments for alopecia areata, a condition that sees the loss of hair all over the body. Eli Lilly and Company, working with Incyte Corporation,…

Continue Reading Baricitinib Granted Breakthrough Therapy Designation for the Treatment of Alopecia Areata
Two Pyoderma Gangraenosum Patients Have Experienced Complete Healing in New Trial

Two Pyoderma Gangraenosum Patients Have Experienced Complete Healing in New Trial

Pyoderma gangraenosum (PG) is a rare auto inflammatory disease which affects the skin. It unfortunately has no currently approved therapies. However, recent preliminary results from a Phase 2a trial have…

Continue Reading Two Pyoderma Gangraenosum Patients Have Experienced Complete Healing in New Trial
Meet Carina Imbrogno, a Rare Artist Living with Ehlers-Danlos Syndrome

Meet Carina Imbrogno, a Rare Artist Living with Ehlers-Danlos Syndrome

I was born in 1974 in Buenos Aires, Argentina to Italian immigrants. I was born with a rare genetic disorder called Ehlers-Danlos syndrome kyphoscoliosis type, but it was not diagnosed…

Continue Reading Meet Carina Imbrogno, a Rare Artist Living with Ehlers-Danlos Syndrome
Dosing Begins in Trial of NLY01, a Treatment of Parkinson’s DIsease

Dosing Begins in Trial of NLY01, a Treatment of Parkinson’s DIsease

An issue with current treatments for Parkinson's disease is that they only provide temporary relief from symptoms. There are no therapies that halt or even slow the progression of the…

Continue Reading Dosing Begins in Trial of NLY01, a Treatment of Parkinson’s DIsease
A First for CRISPR: Editing a Gene in the Retinal Cells of a Leber Congenital Amaurosis Patient

A First for CRISPR: Editing a Gene in the Retinal Cells of a Leber Congenital Amaurosis Patient

  NPR News recently featured an article announcing that scientists at Portland’s Casey Eye Institute have attempted to use CRISPR to edit a gene with the DNA still in place.…

Continue Reading A First for CRISPR: Editing a Gene in the Retinal Cells of a Leber Congenital Amaurosis Patient
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The Mentor She Wished She Had - How Elizabeth Became a Lifeline for EB
Finding Strength Together: Scott and Katie’s Journey with Advanced Kidney
You Are Not Alone: Empowering the Advanced Kidney Cancer Community
Finding Light Through Story-The Power of Ambassadorship in the Endometrial Cancer Community
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