Abstract: The invention features dosing regimens and pharmaceutical formulations for oral administration of palovarotene. The dosing regimens can reduce heterotopic ossification, reduce the number of flare-ups, and/or reduce the severity of flare-ups in subjects suffering from fibrodysplasia ossificans progressiva.

Abstract: The invention features dosing regimens and pharmaceutical formulations for oral administration of palovarotene. The dosing regimens can reduce heterotopic ossification, reduce the number of flare-ups, and/or reduce the severity of flare-ups in subjects suffering from fibrodysplasia ossificans progressiva.

Abstract: The invention describes dosing regimens for treating subjects with fibrodysplasia ossificans progressiva characterized by a quiescent period and a non-quiescent period, wherein a therapeutically effective amount of palovarotene is administered to the subjects during the non-quiescent period, little or no palovarotene is administered to the subjects during the quiescent period, and imatinib is administered to the subjects during the quiescent period and optionally during the non-quiescent period. The dosing regimens can reduce heterotopic ossification, reduce the number of flare-ups, and/or reduce the severity of flare-ups in subjects suffering from fibrodysplasia ossificans progressiva.

Abstract: The invention features methods for inhibiting the formation, reducing the size, and slowing the growth of an osteochondroma in a subject with multiple osteochondroma (MO) by administering to the subject palovarotene (also known as R667), or a pharmaceutically acceptable salt thereof. The methods described herein can also ameliorate complications associated with osteochondroma formation and growth in a subject with MO.

Abstract: The invention features dosing regimens and pharmaceutical formulations for oral administration of palovarotene. The dosing regimens can reduce heterotopic ossification, reduce the number of flare-ups, and/or reduce the severity of flare-ups in subjects suffering from fibrodysplasia ossificans progressiva.

Abstract: The invention features dosing regimens and pharmaceutical formulations for oral administration of palovarotene. The dosing regimens can reduce heterotopic ossification, reduce the number of flare-ups, and/or reduce the severity of flare-ups in subjects suffering from fibrodysplasia ossificans progressiva.

Abstract: The invention features methods for inhibiting the formation, reducing the size, and slowing the growth of an osteochondroma in a subject with multiple osteochondroma (MO) by administering to the subject palovarotene (also known as R667), or a pharmaceutically acceptable salt thereof. The methods described herein can also ameliorate complications associated with osteochondroma formation and growth in a subject with MO.

Abstract: The invention features dosing regimens and pharmaceutical formulations for oral administration of palovarotene. The dosing regimens can reduce heterotopic ossification, reduce the number of flare-ups, and/or reduce the severity of flare-ups in subjects suffering from fibrodysplasia ossificans progressiva.

Abstract: The invention is directed to antisense oligonucleotides that hybridize to the mRNA from a mutant activin A receptor type-1 (ACVR1) gene and inhibit or reduce the expression of the mutant ACVR1 gene. The mutant ACVR1 gene has the mutation c.617G>A. The invention also features pharmaceutical compositions including the antisense oligonucleotides and methods of using the antisense oligonucleotides to treat diseases or conditions (e.g., FOP and DIPG) associated with the expression of the mutant ACVR1 gene.