Common symptoms can include smaller head growth, moderate to severe intellectual disability and developmental delays, the National Organization for Rare Diseases added. Emma said: "We know how ...

A family are fundraising to help support their toddler who has been diagnosed with a rare genetic disorder that affects her ...

Cohorts 1-3 remain free of cytokine release syndrome CRIS-7-derived CD3 design underpins a controlled T-cell response, supporting the differentiated safety profile, combinability with standard of care ...

"Cancer cells from patient's primary tumours that ended up relapsing in the liver or the lung early on adopt cellular states ...

SELLAS’s Phase 2 clinical trial for its investigational drug SLS009 for acute myeloid leukemia showed positive results.

BGB-16673 showed high overall response rates in relapsed/refractory CLL/SLL, with a notable 94.4% response at the 200-mg dose level. Safety analysis revealed that 95.6% of patients experienced ...

Aptevo Therapeutics (APVO) presented preliminary results from its ongoing Phase 1b/2 RAINIER study evaluating mipletamig, a CD123 x CD3 bispecific ...

CLN-049 monotherapy demonstrates promising efficacy, including multiple complete responses and encouraging response durability, in a heavily pretreated all-comer population of patients with R/R AML 31 ...

- Positive 3-month pediatric and 18-month adult clinical data from OPGx-LCA5 Phase 1/2 trial support the potential for restoring cone-mediated vision - - Successful FDA RMAT meeting provides the ...

Obe-cel demonstrates high remission rates in pediatric patients with high-risk r/r B-ALL with overall response rate (ORR) of 95.5%; low rates of ...

The Nazi dictator had Kallmann syndrome, which can affect the size and development of sex organs Toria Sheffield joined the PEOPLE editorial staff in 2024. Her work as a writer/editor has previously ...