Santhera Pharmaceuticals’ Agamree (vamorolone) has been accepted by the Scottish Medicines Consortium (SMC) to treat Duchenne ...

Santhera Pharmaceuticals will launch its new drug for Duchenne muscular dystrophy in the UK within the next few weeks, after ...

NICE’s positive final guidance for Agamree follows confirmation that no appeals were submitted against its positive December ...

Santhera has begun launch preparations for AGAMREE® in UK with first sales expected in this quarterPratteln, Switzerland, January 16, 2025 – ...

Duchenne muscular dystrophy (DMD), a rare condition in females, causes muscle weakness. 27-year-old Stephanie, who was ...

An Irish woman has launched a fundraiser to help raise money for medical treatment for her seven-year-old son. Una Ennis' son ...

Of the 24 drugs, three approvals deviated from the others -- aducanumab; the synthetic hormone hydroxyprogesterone caproate ...

The FDA established its accelerated approval program in 1992 to allow drugs to reach the market quickly for life-threatening ...

The landscape of rare diseases is constantly changing as new ailments and conditions are identified and reported regularly.

AbbVie and Gilead are going back to their roots and leaning on their established areas of expertise to set themselves up for ...

With the use of deep learning and advanced algorithms, generative AI can promote acceleration in the design and development ...

Regenxbio's rolling BLA submission completion of RGX-121 for the treatment of patients with MPS II expected in Q1 of 2025.