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The Drug Burosumab Demonstrates Long-Term Benefits for X-Linked Hypophosphatemia

The Drug Burosumab Demonstrates Long-Term Benefits for X-Linked Hypophosphatemia

According to a story from Medscape, the drug burosumab (marketed as Crysvita) demonstrated its ability to provide long term benefits for nearly two years to patients with the rare disease…

Continue Reading The Drug Burosumab Demonstrates Long-Term Benefits for X-Linked Hypophosphatemia
IMFINZI Shows Efficacy for Stage IV Non-Small Cell Lung Cancer

IMFINZI Shows Efficacy for Stage IV Non-Small Cell Lung Cancer

According to a story from us.acrofan.com, the biopharmaceutical company AstraZeneca recently announced the release of positive results for a clinical trial testing the company's drug durvalumab (marketed as IMFINZI) in…

Continue Reading IMFINZI Shows Efficacy for Stage IV Non-Small Cell Lung Cancer
The AI Revolution in Rare Disease Drug Discovery

The AI Revolution in Rare Disease Drug Discovery

A recent announcement by Atomico, one of Europe’s largest venture capital firms based in London, introduced its new partner Healx. The startup can boast of having the world’s most comprehensive…

Continue Reading The AI Revolution in Rare Disease Drug Discovery
Editor’s Choice: Monster Mashing, Rare Disease Smashing

Editor’s Choice: Monster Mashing, Rare Disease Smashing

Boo! We hope everyone's trick or treat bag was heavy on Reese's this year! This week we're highlighting stories about a youth advocate, gene therapy, and a multiple sclerosis predictor.…

Continue Reading Editor’s Choice: Monster Mashing, Rare Disease Smashing
Preclinical Findings for Phenylketonuria and Metachromatic Leukodystrophy Gene Therapies Look Promising

Preclinical Findings for Phenylketonuria and Metachromatic Leukodystrophy Gene Therapies Look Promising

According to a story from BioSpace, the genetic medicines company Homology Medicines, Inc., has recently released preclinical data supporting the development of two experimental gene therapies. One, called HMI-202, is…

Continue Reading Preclinical Findings for Phenylketonuria and Metachromatic Leukodystrophy Gene Therapies Look Promising
An Experimental Treatment for Growth Hormone Deficiency Performs Well in Phase 3 Trial

An Experimental Treatment for Growth Hormone Deficiency Performs Well in Phase 3 Trial

According to a story from globenewswire.com, the experimental drug somatrogon, which is being co-developed by Pfizer, Inc. and OPKO Health, Inc., was able to achieve its primary endpoint in a…

Continue Reading An Experimental Treatment for Growth Hormone Deficiency Performs Well in Phase 3 Trial
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
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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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