Science Could a drug already available for cancer be used to treat some children with Noonan syndrome? This is what a ...

The worlds that I’m creating with the detective novels and with 'Learwife' are different. They’re going to do very different ...

BioMarin's gene therapy pipeline has had setbacks, including a three-year delay in US approval of Roctavian and stalled development of PKU gene therapy BMN 307 as the Food and Drug Administration ...

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) reported fourth-quarter adjusted EPS of 92 cents, up from 49 cents reported a year ago, beating the consensus of 53 cents. The company reported sales of $747 ...

Parents Hailey and Juan Lucero said they knew what to expect having their daughter, Adaliyah as a preemie, but what they did ...

Government of India has provided tax relief on life-saving drugs and medicines used for rare diseases. As per the Ministry of Finance’s response in Rajya Sabha on February 11, 2025, basic customs duty ...

The mother at the centre of Melanie Reid’s latest podcast investigation is set to be deported in the coming weeks, despite ...

Outside of work, Laura spends her time on humor writing, metalsmithing and bravely battling a case of late-onset imposter syndrome. Laura earned a master's in international affairs from Columbia ...

Suzhou Genhouse Bio Co. Ltd. has disclosed tyrosine-protein phosphatase non-receptor type 11 (PTPN11; PTP-2C; SHP-2) inhibitors reported to be useful for the treatment of cancer, Noonan syndrome and ...

CHARGE syndrome in two; trisomy 21; Noonan syndrome; and infantile spinal muscular atrophy), two in group 3 (Rubinstein Taybi syndrome; Ivemark syndrome) and two in group 4 (trisomy 21 ...