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Their Daughter Was Maturing Slowly. Then, She Began to Regress.

Their Daughter Was Maturing Slowly. Then, She Began to Regress.

As originally reported in SF Gate, Jeannette and Victor Vega quickly realized that taking care of their second daughter, Tiana, was not going to be so easy this time. From…

Continue Reading Their Daughter Was Maturing Slowly. Then, She Began to Regress.
Experimental Non-Steroid Treatment Displays Potential for Congenital Adrenal Hyperplasia

Experimental Non-Steroid Treatment Displays Potential for Congenital Adrenal Hyperplasia

According to a story from Healio, the results from a recent phase 2A clinical trial have demonstrated the effectiveness of an experimental drug in treating congenital adrenal hyperplasia (CAH). The…

Continue Reading Experimental Non-Steroid Treatment Displays Potential for Congenital Adrenal Hyperplasia
A Drug Used to Treat Psoriasis Could be Effective for Ulcerative Colitis

A Drug Used to Treat Psoriasis Could be Effective for Ulcerative Colitis

According to a story from Medical News Bulletin, a recent study that was first published in the New England Journal of Medicine appears to have revealed a promising new treatment for ulcerative…

Continue Reading A Drug Used to Treat Psoriasis Could be Effective for Ulcerative Colitis
Moxibustion May Provide Short-Term Benefits for Ankylosing Spondylitis

Moxibustion May Provide Short-Term Benefits for Ankylosing Spondylitis

According to a story from Ankylosing Spondylitis News, a recent study has found that moxibustion, a form of heat therapy, could provide short-term benefits for patients with the rare disease…

Continue Reading Moxibustion May Provide Short-Term Benefits for Ankylosing Spondylitis
Researchers are Hopeful a New Gene Therapy Could Cure Canavan Disease

Researchers are Hopeful a New Gene Therapy Could Cure Canavan Disease

Gene therapies have huge potential in the field of rare disease. The fact that these therapies could offer a potential cure otherwise fatal diseases has given families incredible hope. Unfortunately,…

Continue Reading Researchers are Hopeful a New Gene Therapy Could Cure Canavan Disease
New Data Highlights Zolgensma’s Benefits for Spinal Muscular Atrophy Patients

New Data Highlights Zolgensma’s Benefits for Spinal Muscular Atrophy Patients

According to a story from finanznachrichten.de, the latest data from three phase 3 studies is giving further confirmation to the capability of the recently approved gene therapy Zolgensma to have…

Continue Reading New Data Highlights Zolgensma’s Benefits for Spinal Muscular Atrophy Patients
Editor’s Choice: Aries Advocacy and Rare Sibling Bonds

Editor’s Choice: Aries Advocacy and Rare Sibling Bonds

Happy Thursday everyone! This week, we're sharing a video that will warm your heart, as well as an article about how to improve care in older Multiple Myeloma patients. We…

Continue Reading Editor’s Choice: Aries Advocacy and Rare Sibling Bonds
ICYMI: FDA approves Pexidartinib (Turalio): A Drug for Rare Joint Tumor

ICYMI: FDA approves Pexidartinib (Turalio): A Drug for Rare Joint Tumor

According to the American Journal of Managed Care (AJMC), the US Food and Drug Administration (USFDA) has approved Pexidartinib (Turalio) drug as first targeted therapy to treat symptomatic Tenosynovial Giant…

Continue Reading ICYMI: FDA approves Pexidartinib (Turalio): A Drug for Rare Joint Tumor
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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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