People say fatherhood is not for cowards. When three Malaysians found themselves gifted with special children, they could have chosen denial, indifference or escape. Instead, they stepped up to the plate. TWO people fall madly in love, get married, then start a family. They embrace parenthood with delight and watch...
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HENDERSON, Nev. – Spectrum Pharmaceuticals, Inc. (NasdaqGS: SPPI), a biopharmaceutical company focused on novel and targeted oncology therapies, announced the presentation of safety and efficacy results from Cohort 4 of the ZENITH20 clinical trial. This data is from 48 first-line patients with non-small cell lung cancer (NSCLC) with HER2 exon...
Urbana, IL – The Speech Accessibility Project has expanded its recruitment and is inviting U.S. and Puerto Rican adults living with amyotrophic lateral sclerosis to participate. Those interested in participating can sign up online. Funded by Big Tech companies Amazon, Apple, Google, Meta, and Microsoft, the University of Illinois Urbana-Champaign aims...
GURNEE — Dylan Mudrick looks like other 15-month-old babies. His blue eyes light up when he smiles, and he is at a healthy weight for his age. However, Dylan was diagnosed with a rare disease when he was 4 days old and is now waiting for a liver transplant. When...
STAMFORD, Conn. — SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, announced today that the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for nirogacestat, an oral gamma secretase inhibitor, for the treatment of adults with desmoid tumors. If...
STAMFORD, Conn. — SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, announced today that the U.S. Food and Drug Administration (FDA) has approved OGSIVEO™ (nirogacestat), an oral gamma secretase inhibitor, for the treatment of adult patients with progressing desmoid tumors who require...
Stamford, Conn. — SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, announced that data from the Phase 3 DeFi trial of nirogacestat, an investigational oral gamma secretase inhibitor, in adult patients with progressing desmoid tumors, were...
SOUTH SAN FRANCISCO, Calif. — Spruce Biosciences, Inc., a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for rare endocrine disorders with significant unmet medical need, today announced completion of enrollment in its CAHmelia-204 clinical trial of tildacerfont for the treatment of adult classic congenital adrenal hyperplasia (CAH)....
ALISO VIEJO, Calif. — SpyGlass Pharma™, a privately-held ophthalmic biotechnology company, announced today 1-year follow up data from a first-in-human study of 23 patients with glaucoma or ocular hypertension implanted with SpyGlass’ Intraocular Lens (IOL) based Drug Delivery Platform with bimatoprost at the time of cataract surgery. These data will...
Mill Valley, CA – SRF has established a dedicated account to support research regarding SYNGAP1 missense variants. This account is being launched with a $10,000 donation from Dennis and Janet Nordmoe. This is a cause close to their hearts, as their granddaughter Olivia was diagnosed with SYNGAP1-Related Disorder (SRD) caused...