UC Davis Health is conducting a clinical trial combining fetal surgery with stem cells to treat spina bifida, offering new hope for affected families.

Protalix BioTherapeutics' PRX-115, a PEGylated uricase, shows promising dose-dependent urate-lowering effects in a Phase 1 study for individuals with gout.

Lupin has been granted tentative FDA approval for its generic version of Janumet (sitagliptin and metformin hydrochloride) tablets, used to manage type 2 diabetes.

Japan's health ministry panel has approved tofersen, a drug developed by Biogen, for treating amyotrophic lateral sclerosis (ALS) patients with SOD1 gene mutations.

Windtree Therapeutics has engaged New Growth Advisors to explore strategic options for its cardiovascular portfolio, including istaroxime, following positive Phase 2 results.

A group of leading researchers is advocating for the FDA to prioritize amyloid clearance as the primary criterion for approving Alzheimer's drugs.

Matica Biotechnology, a subsidiary of CHA Biotech, has partnered with Treovir to manufacture herpes simplex virus (HSV) vectors for pediatric brain tumor therapy.

Relmada Therapeutics discontinues Phase 3 trials (RELIANCE II and RELIGHT) of REL-1017 for depression after unfavorable data monitoring committee evaluation.

A recent study indicates that low-dose oral minoxidil (LDOM) is a safe and effective treatment for hair loss in women undergoing breast cancer treatment.

The PARASOL initiative analyzed 1600 FSGS patients to identify reliable surrogate endpoints for clinical trials, addressing the challenge of long follow-up periods.

A clinical trial reveals that selinexor, an FDA-approved drug, effectively shrinks tumors in non-small cell lung cancer (NSCLC) patients with KRAS mutations.

Single-cell RNA sequencing reveals that biologic therapies alter the transcriptional states of circulating immune cells in severe eosinophilic asthma patients.

DiagnaMed Holdings Corp. has received FDA Orphan Drug Designation for molecular hydrogen as a potential treatment for ALS, offering key development incentives including seven years of market exclusivity.

Recordati has secured worldwide rights to Enjaymo, the first and only approved treatment for cold agglutinin disease (CAD), from Sanofi for an upfront payment of $825 million with potential milestone payments of $250 million.

The UK government has introduced legislation to ban xylazine, known as the "zombie drug," and 21 other substances as part of efforts to combat drug-related deaths and disrupt criminal gangs.

The NCCN has issued significant updates across 20 different cancer types in 2024, including new tumor-agnostic approvals for repotrectinib and trastuzumab deruxtecan for solid tumors with specific genetic markers.

Sezaby, a preservative-free phenobarbital formulation by Sun Pharmaceutical Industries, has been approved as the first FDA-sanctioned treatment specifically for neonatal seizures in both term and preterm infants.

Black women represent only 5.4% of participants in recent triple-negative breast cancer clinical trials, despite being twice as likely to be diagnosed with the disease compared to other racial groups.

Clinical trial terminations in CNS drug development have reached a concerning peak of 1,488 in 2022, with Phase II trials experiencing the sharpest increase from 204 to 650 terminations between 2014-2022.

The FDA has released comprehensive guidance clarifying the use of electronic systems, e-records, and e-signatures in clinical investigations, emphasizing data integrity and security through enhanced controls and audit trails.