This International Day of Persons with Disabilities IDPD know more about Achondroplasia Also find out about its cure ...

placebo-controlled pivotal study for the treatment option for children with achondroplasia; and BBP-631, an AAV5 gene ...

BridgeBio secured approval for Attruby Friday, along with the all-important mortality benefit that could give the drug a ...

Biomarin has a near $3bn per annum drug franchise, and is profitable. Elliott Investment Management may be attempting to ...

An injection given during some asthma and COPD attacks is more effective than the current treatment of steroid tablets, reducing the need for further treatment by 30%. UCSF Benioff Children's ...

TTR stabilizer in clinical development, lived longer and better as shown in the ATTRibute-CM study. This is the only Phase 3 ...

For larger or more noticeable scars, there are some treatments that can help reduce their size or make them less noticeable. Scarring is a natural part of the healing process after an injury.

Image: Professor Ravi Savarirayan The study, sponsored by BridgeBio Pharma Inc, involved 72 children with achondroplasia from Australia, the UK, the US, Spain, France and Canada. It reported the ...

A promising daily tablet is effective at increasing height and improving proportional limb growth in children with achondroplasia, the most common form of dwarfism, according to a new study. And the ...

Relative to an untreated achondroplasia reference population ... the mean change from baseline was −0.12. "Additional treatment duration will be required to determine whether the improvement ...

Relative to an untreated achondroplasia reference population, the mean change from baseline in height z score was 0.54 at 18 months; in the upper-to-lower body segment ratio, the mean change from ...