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- PMLiVE

Isis Pharmacueticals

- PMLiVE

Sanofi expands rare disease pipeline with $2.2bn Inhibrx acquisition

The deal includes a mid-stage candidate for alpha-1 antitrypsin deficiency

Patient Advocate Engagement: A Customer Story

Learn how our client executed a series of 3 virtual touchpoints with 5 rare disease patient advocates to:- Develop a better understanding of the current treatment landscape and lived patient...

Impetus Digital

- PMLiVE

MHRA approves UCB’s Zilbrysq as generalised myasthenia gravis add-on therapy

The autoimmune disease has a global prevalence of 100 to 350 cases per every one million people

- PMLiVE

Takeda’s HyQvia granted FDA approval to treat rare neuromuscular disorder CIDP

Approximately five to seven people per 100,000 in the US are affected by the disorder

- PMLiVE

Roche’s subcutaneous Tecentriq granted EC approval for multiple cancer types

The formulation reduces treatment time by around 80% compared to intravenous infusion

- PMLiVE

New antibiotic compound could potentially treat lethal hospital infections

Zosurabalpin successfully eliminated the multi-resistant A baumannii bacteria

On a journey to diverse, inclusive, and more efficient rare disease trials

Although orphan drugs represent a sizeable portion of new drug approvals, most rare diseases still lack effective treatment. Even more so than for other conditions, rare disease management contains large care...

Impetus Digital

- PMLiVE

Study programme to investigate blocking brain inflammation in Parkinson’s patients

If successful, the findings could lead to new medicines to treat the neurological disease

- PMLiVE

AstraZeneca and Ionis’ Wainua granted FDA approval for rare disease ATTRv-PN

ATTRv-PN leads to peripheral nerve damage with motor disability within five years of diagnosis

- PMLiVE

Roche shares positive late-stage results for inavolisib combination in advanced breast cancer

About 290,000 people in the US will be diagnosed with breast cancer in 2023

- PMLiVE

Roche to acquire Carmot Therapeutics in deal worth up to $3.1bn

The company will gain access to a portfolio of incretin-based therapies for diabetes and obesity

- PMLiVE

Merck to acquire Caraway Therapeutics in a deal worth up to $610m

The deal gives Merck access to a pipeline of therapeutics for neurodegenerative and rare diseases

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