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My sore throat turned out to be a brain tumor: Girl, 19, describes shock diagnosis that required innovative new surgical technique to remove the lump

For years, Christina Giuffrida had been experiencing an on-again, off-again sore throat. The 19-year-old from Yonkers, New York, also had slight balance issues and would struggle to hear people speaking at normal volumes. One doctor told her and her parents, it was a problem with her eyesight. Another said it was a flare-up from a previous mononucleosis infection or allergies.

It’s time to find new targets for brain diseases instead of just pursuing old ones

Ifirst learned about drug discovery and development in the mid-1990s when two of my sons were diagnosed with a brutal, untreatable genetic disease called ataxia-telangiectasia (A-T). Children with A‑T struggle with immune deficiency and lung problems, and have an extremely high cancer risk.

Check-Cap Goes Global with Colorectal Cancer Diagnostic Device

If you know that there’s a way to detect cancer early, would you get screened? Many people would say yes, but statistics disagree. About 40 percent of Americans do not get screened for colorectal cancer because they refuse to get or cannot get a colonoscopy. According to the American Cancer Society, 51,000 people in the US are estimated to die from colorectal cancer this year.

With phase 2 update, Krystal Biotech eyes phase 3 for rare skin disease gene therapy

Krystal Biotech unveiled data from a small phase 2 study showing its topical gene therapy closed the majority of wounds in patients with a rare skin disorder. The company is waiting on data for how long the wounds stayed closed after treatment before it moves the program into phase 3. It expects to start the pivotal trial by the end of the year.

Gene therapy biotech sees its stock rocket higher on promising results for rare cases of butterfly disease

Shares of Krystal Biotech took off this morning $KRYS after the little biotech reported promising results from its gene therapy to treat a rare skin disease called epidermolysis bullosa.

First Patient Dosed in Phase 3 Trial of Etrasimod for Moderate to Severe UC

The Phase 3 trial ELEVATE UC 52 evaluating Arena Pharmaceuticals’ investigational therapy etrasimod for patients with moderately to severely active ulcerative colitis has dosed its first patient. “We are pleased to enroll the first patient in the ELEVATE UC trial, supporting etrasimod’s potential as an important future therapy for ulcerative colitis,” Darshan Anandu, MD, of G.I. Specialists of Houston, said in a press release.

With $45M Nurix Deal, Gilead Enters Crowded Protein Degradation Field

Gilead Sciences is turning to Nurix Therapeutics in an effort to discover new drugs that harness the cellular machinery our bodies use to dispose of damaged or harmful proteins.

SanFranBusTimes

Why Gilead could pay more than $2 billion in deal with this small S.F. biotech

Founded by three professors from UC Berkeley and UCSF, Nurix’s protein-degradation approach to cancer, and potentially other diseases, could stop cancer in its tracks.