The effects of PBI-4050 demonstrated in animal models have been replicated in Phase 2 studies in idiopathic pulmonary fibrosis (IPF), in metabolic syndrome with type 2 diabetes and in Alstrom syndrome
. PBI-4050 is entering pivotal placebo-controlled Phase 3 clinical trials for the treatment of IPF and has already begun placebo- controlled Phase 2 trials in metabolic syndrome and type 2 diabetes patients.
Theory-of-mind in adolescents and young adults with Alstrom syndrome
. International Journal of Pediatric Otorhinolaryngology, 78(3), 530-536.
: intrafamilial phenotypic variability in sibs with a novel nonsense mutation of the ALMS1 gene.
Now, with her son suffering from all the classic symptoms of Alstrom Syndrome
, including a heart condition, sight and hearing loss, plus liver problems and diabetes, she can't help but fear the same thing happening to her son.
Marshall, who created and led Alstrom Syndrome
In conjunction, the company has received the US FDA's Rare Pediatric Disease Designation as well as the US FDA's and the EMA's Orphan Drug Designation for PBI-4050 for the treatment of Alstrom syndrome
, a rare inherited autosomal recessive syndrome characterized by the onset of obesity in childhood or adolescence, Type 2 diabetes, often with severe insulin resistance, dyslipidemia, hypertension and severe multi-organ fibrosis involving the liver, kidney and heart.
ProMetic Life Sciences' orally active lead drug candidate, PBI-4050, has been granted an orphan drug designation status for the treatment of Alstrom Syndrome
("AS") by the European Commission.
INTRODUCTION: Alstrom Syndrome
is a rare genetic disorder, inherited as autosomal recessive.
Simmy was diagnosed with Alstrom Syndrome
, a rare genetic condition which affects eyesight, as well as hearing and internal organs, when she was seven years old.
Rhythm Pharmaceuticals announced that updated data from the company's ongoing Phase 2 basket studies evaluating setmelanotide in people with Bardet-Biedl Syndrome, or BBS and Alstrom Syndrome
are being presented at the 57th Annual European Society for Paediatric Endocrinology, or ESPE.
Biopharmaceutical company Prometic Life Sciences Inc (TSX:PLI) (OTCQX:PFSCF) reported on Tuesday the receipt of US Food and Drug Administration (FDA) Rare Pediatric Disease Designation for its PBI-4050 small molecule drug candidate for the treatment of Alstrom syndrome
Born with a rare genetic condition called Alstrom syndrome
, avid Doctor Who fan Mr Parry, who lives with his parents in Ormesby, is deaf, blind and diabetic.
The research will be managed by Alstrom Syndrome
UK support group but carried out by Birmingham Children's Hospital and Torbay Hospital, in Devon, along with Cambridge University.
The Company is also evaluating setmelanotide in four additional rare genetic disorders of obesity, including Bardet-Biedl Syndrome, or BBS, Alstrom Syndrome
, POMC and other MC4R pathway heterozygous deficiency obesities, and POMC epigenetic disorders.
Born with an extremely rare genetic condition called Alstrom syndrome
, avid Dr Who fan Mr Parry, who lives with his parents in Ormesby, is deaf, blind and diabetic.