Sen. Ron Johnson (R-Wisc.) is speaking out on the matter of PTC Therapeutics Inc.’s Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (DMD). Warren, N.J.-based PTC disclosed Feb. 12 that it has withdrawn the NDA resubmission for approval of the protein restoration therapy after regulators said the package was “unlikely to meet the agency's threshold of substantial evidence of effectiveness.” Johnson stepped into the controversial DMD space with a news conference March 10, decrying the loss of compassionate-use access to Translarna, which has been helping patients.

Hightide wins China NDA acceptance for oral metabolic therapy

China’s National Medical Products Administration (NMPA) has accepted for review Hightide Therapeutics Inc.’s NDA for HTD-1801 for type 2 diabetes, marking the Shenzhen-based company’s first NDA submission and a major step toward commercialization. The filing positions Hightide among the next wave of metabolic‑disease developers targeting the emerging cardiovascular‑kidney‑metabolic spectrum, an area increasingly viewed as the future of diabetes care.

Three pediatric brain cancer types share a pineal gland origin

Similarities among three pediatric brain tumors that arise in different structures of the CNS – pineoblastoma (PB), retinoblastoma (RETB) and Group 3 medulloblastoma (G3-MB) – have been linked to their shared origin during pineal gland development. Scientists at St. Jude Children’s Research Hospital have identified the molecular signatures that drive these tumors from pinealocyte progenitor cells that conserve a common differentiation program, providing a shared therapeutic target for these three cancer types. “Even though they are different tissues, different lineages, they are in many ways sharing the same transcription factors and programs that are likely contributing to a proliferative state,” senior author Paul Northcott, director of the Center of Excellence in Neuro-Oncology Sciences (CENOS) at St. Jude Children’s Research Hospital, told BioWorld.

Samsung is adding trials and EHR access to Galaxy devices

Samsung Electronics Co. Ltd. is partnering with Verily Life Sciences LLC, an Alphabet Inc. company, and B.well Connected Health to turn Samsung Galaxy phones and smart watches into the “front door” of U.S. health care. The back-to-back deals with Verily and B.well, announced at HIMSS26 (Healthcare Information and Management Systems Society 2026 Global Health Conference and Exhibition) in Las Vegas, adds to big tech’s push to unite consumer electronics with health care and medicine.

China’s Qyuns moves IL-17 biologic toward first approval

Qyuns Therapeutics Co. Ltd. has moved closer to its first commercial product after China’s National Medical Products Administration (NMPA) accepted its NDA for IL-17 antibody crusekitug (QX-002N) for treating ankylosing spondylitis, a chronic inflammatory disease that affects the spine and sacroiliac joints. The milestone marks the first innovative drug developed by the Shanghai‑based biotech to reach the NDA stage and reflects growing momentum across its autoimmune pipeline.

ARPA-H sets sights on next-gen biosensors

The Advanced Research Projects Agency for Health (ARPA-H) launched its Delphi program this week to advance the development of the next generation of wearable and ingestible biosensors that can securely report deep biological data to optimize care. Typically focusing on a single component of health monitoring, such as heartrate or blood glucose, current biosensors miss “rich data streams that can prevent life-threatening health crises and help patients better manage chronic conditions, safely recover from illness outside of a hospital and improve overall wellness,” the agency said. By reimagining that design with modular “chiplet” technology, the Delphi program is expected to pave the way for more affordable, precise biosensor devices that can monitor hormones, inflammatory markers and other important biochemical signals.

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