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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…

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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.…

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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…

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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…

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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…

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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
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